Search Result for Epithelium

List View
Table View

125 results for Epithelium

Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia. - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Delivery of GFP via shuttle peptides to mouse airway epithelium via nasal instilation. --> Delivery of GFP via shuttle peptides to mouse airway epithelium via nasal instilation. --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium McCray Paul B Delivery of GFP via shuttle peptides to mouse airway epithelium via nasal instilation. Delivery efficiency was quantified in large and small airways by counting the number of GFP positive cells divided by the number of DAPI cells. Projects Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Alt-R® S.p. Cas9 Nuclease V3 - Genome Editor [Delivery Systems Initiative] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
g-loxP2_C9 - Guide [Delivery Systems Initiative] - [In Vivo] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium This sgRNA targets the Ai9 and related transgenes Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
BALB/c mouse - Model System [Delivery Systems Initiative] - [In Vivo] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium BALB/cJ is a commonly used inbred. Key traits include a susceptibility to developing the demyelinating disease upon infection with Theiler's murine encephalomyelitis virus. The BALB/cJ substrain is susceptible to Listeria, all species of Leishmania, and several species of Trypanosoma, but is resistant to experimental allergic orchitis (EAO). Experiments Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
Matched on: --> Epithelium of bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium Other Id: Santa Cruz, SC-25555 Anti-CC10 (Rabbit) Polyclonal Antibody, dilution used 1:2,000
AsCas12a (IDT and Feldan Therapeutics) - Genome Editor [Delivery Systems Initiative] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium McCray Paul B In vivo shuttle peptide delivery of Cas9 and Cas12a RNPs in mouse airway epithelia. Gene editing was quantified by the GFP+ cells in large and small airways following 1 delivery of GFP protein by GFP positive cells compared to DAPI stained cells. Projects Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
GFP-NLS - Genome Editor [Delivery Systems Initiative] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium Nuclear targeted GFP Experiments Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
sgAi9L - Guide Matched on: --> Epithelium of bronchus Epithelium of main bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium This sgRNA targets the Ai9 and related transgenes
sgAi9R - Guide Matched on: --> Epithelium of bronchus Epithelium of main bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium This sgRNA targets the Ai9 and related transgenes
mTmG mouse - Model System [Delivery Systems Initiative] - [In Vivo] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium ROSAmT/mG is a cell membrane-targeted, two-color fluorescent Cre-reporter allele. Prior to Cre recombination, cell membrane-localized tdTomato (mT) fluorescence expression is widespread in cells/tissues. Cre recombinase expressing cells (and future cell lineages derived from these cells) have cell membrane-localized EGFP (mG) fluorescence expression replacing the red fluorescence Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
Cre recombinase - Genome Editor [Small Animal Testing Center (SATC), Delivery Systems Initiative] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Epithelium of mucosa Epithelium Endo-epithelium Lung epithelium Meso-epithelium Cre recombinase delivered by AAV (see vector details) Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
ssAAV5-sgB.saCas9 - Vector [Small Animal Testing Center (SATC)] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Epithelium of mucosa Columnar epithelium Epithelium Endo-epithelium Lung epithelium Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
BCM_ssAAV5-Sa_sgB - Guide [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Epithelium of mucosa Columnar epithelium Epithelium Endo-epithelium Lung epithelium This gRNA targets the Ai9 and related transgenes Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Matched on: --> Epithelium of main bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium Other Id: Rockland Cat# 600-401-379 Anti-RFP (RABBIT) Antibody
Matched on: --> Epithelium of bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium Other Id: Thermo Fisher Scientific, MA5-15257 Anti-RFP (Mouse) Monoclonal Antibody, dilution used 1:300
Matched on: --> Epithelium of bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium Other Id: Rockland, 600- 401-379 Anti-RFP (Rabbit) Polyclonal Antibody
Matched on: --> Epithelium of bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium Other Id: Santa Cruz, SC-23950 Anti-alpha Tubulin (Mouse) Monoclonal Antibody, dilution used 1:200
SpCas9 - Genome Editor [Biological Systems] Matched on: --> Epithelium of bronchus Epithelium of main bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium HA-SV40NLS-SpCas9-SV40NLS Experiments A novel human T cell platform to define biological adverse effects of genome editing
AAV.pU1a-SpCas9 - Vector Matched on: --> Epithelium of bronchus Epithelium of main bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) - Vector Matched on: --> Epithelium of bronchus Epithelium of main bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP
g-loxPbot_C12a - Guide [Delivery Systems Initiative] - [In Vivo] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Lung epithelium Epithelium of mucosa Epithelium Foregut epithelium Endo-epithelium This sgRNA targets the Ai9 and related transgenes at two sites Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
SaCas9 - Genome Editor [Small Animal Testing Center (SATC)] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Epithelium of mucosa Columnar epithelium Epithelium Endo-epithelium Lung epithelium Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
S10 - Delivery System [Delivery Systems Initiative] - [In Vivo, In Vitro] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Epithelium of mucosa Columnar epithelium Epithelium Endo-epithelium Lung epithelium Shuttle peptide used to deliver reagents to airway epithelia Experiments Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia. Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia Peptide Shuttle optimization to deliver Cas9 RNP to human airway epithelia cells Gene editing in vitro by various peptide variants delivering Cas RNPs to primary Human airway epithelia cells. Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA using the S10 shuttle peptide to activate the mTmG reporter in mouse brain Peptide Shuttle optimization to deliver Cas12a RNP to human airway epithelia cells Gene editing in vitro by various peptide variants delivering Cas12a in NK cells.
scAAV5-Cre-GFP - Vector [Small Animal Testing Center (SATC)] Matched on: --> Epithelium of bronchiole Epithelium of main bronchus --> Epithelium of mucosa Columnar epithelium Epithelium Endo-epithelium Lung epithelium Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Matched on: --> Epithelium of main bronchus --> Columnar epithelium Epithelium Foregut epithelium Ciliated epithelium Endo-epithelium Other Id: Cell Signaling Technology Cat# 2956 GFP (D5.1) XP Rabbit mAb antibody
D10 - Delivery System [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium Experiments Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Ai9 mouse - Model System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo] Matched on: --> Epithelium of bronchus Epithelium of main bronchus --> Epithelium of mucosa Ecto-epithelium Epithelium Endo-epithelium Meso-epithelium Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Cre Recombinase dose escalation study in Ai9 mice Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
D237 - Delivery System [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium Experiments Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
SpCas9 - Genome Editor [Small Animal Testing Center (SATC)] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
BCM_ssAAV5-Sp_sgB - Guide [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium This sgRNA targets the Ai9 and related transgenes Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
SpyCas9 g-loxP2_C9 - Guide [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium This sgRNA targets the mTmG transgene Experiments Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
ssAAV5-sgA+sgB-U1A.GFP - Vector [Small Animal Testing Center (SATC)] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Ai9 mouse (BCM) - Model System [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Ecto-epithelium Lung epithelium Epithelium Endo-epithelium Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Ai9-SauSpyCas9 mouse - Model System [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium Ai9-SauSpyCas9 is a version of Ai9 that has a single guide RNA sites on both sides of the STOP cassette. This is a Cre reporter allele that has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Ai9 mice express robust tdTomato fluorescence following Cre-mediated recombination. Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
[Validation] Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides - Experiment [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium Heaney Jason D Ribonucleoproteins for CRISPR/Cas9 editing are complexed with amphiphilic peptides for delivery to lung airway epithilia via intranasal instillation into mTmG reporter mice. Editing is detected by production of GFP protein, and green fluorescence in airway linings Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - McCray Validation (McCray Jr., Paul)
ssAAV5-spCas9 - Vector [Small Animal Testing Center (SATC)] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
BCM_ssAAV5-Sp_sgA - Guide [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium This sgRNA targets the Ai9 and related transgenes Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
mTmG mouse (congenic) - Model System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Ecto-epithelium Lung epithelium Epithelium Endo-epithelium mTmG is a double-fluorescent reporter transgenic mouse which expresses membrane-targeted tdTomato flanked by loxP sequences, followed by membrane-targeted GFP. After genomic cleavage by Cas9 at two sites, or Cre recombinase between loxP sites, tdTomato expression is lost and GFP is expressed. Experiments Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA using the S10 shuttle peptide to activate the mTmG reporter in mouse brain Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain
[Validation] Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice - Experiment [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Epithelium of main bronchus Epithelium of bronchiole --> Epithelium of mucosa Lung epithelium Epithelium Endo-epithelium Meso-epithelium Heaney Jason D AAV5 encoding CRISPR/Cas editing machinery were delivered to the lungs of reporter mice by intratracheal instillation. After 4 weeks incubation, the mice were dissected and the lungs imaged for the presence of tdTomato fluorescence, indicating successful editing. Editing calculated by dividing the number of tdTomato+ red cells by the number of nuclei in each airway Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Gao Validation (Gao, Guang-Ping )
AAV - Delivery System [Delivery Systems Initiative] - [In Vivo] Matched on: --> Digestive tract epithelium Columnar epithelium Epithelium Unilaminar epithelium Endo-epithelium See vector details Experiments Cre Recombinase dose escalation study in Ai9 mice
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector. - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium Asokan Aravind A dual vector strategy was employed: one self complementary vector delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (single stranded vector). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=4) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:1 ratio of cas9 to guide RNA (2e12vg of CMV Sacas9 vector and 2e12vg of the self complementary sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
SaCas9-Lagor - Genome Editor [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
AAV9-Ai9-sgRNA1 + sgRNA2 - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
AAVcc47_pTR_self comp 2xU6-Ai9 guides - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
113-O12B - Delivery System [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Unilaminar epithelium combinatorial library cationic lipid nanoparticles Experiments Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Asokan Aravind C57/BL6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding an mCherry reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
[Validation] Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. - Experiment [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Columnar epithelium Foregut epithelium Epithelium Endo-epithelium Meso-epithelium Heaney Jason D Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues. Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Asokan Validation (Asokan, Aravind)
SauCas9 - Genome Editor [Small Animal Testing Center (SATC)] Matched on: --> Columnar epithelium Foregut epithelium Epithelium Endo-epithelium Meso-epithelium Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Ai9 SaCas9 Guide B - Guide [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Columnar epithelium Foregut epithelium Epithelium Endo-epithelium Meso-epithelium This gRNA targets the Ai9 and related transgenes Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
STS159 (mTmG; Sp_t2:Sp_c20_mTmG) - Guide [Delivery Systems Initiative] - [In Vivo, In Vitro] Matched on: --> Ecto-epithelium Epithelium Targets mTmG and related reporter transgenes Experiments Testing newly chemically modified crRNA and tracrRNA in mTmG mouse embryonic fibroblasts Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center
RNP-NC-RVG - Delivery System [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium The nanocapsule is a thin glutathione (GSH)-cleavable covalently crosslinked polymer coating around a preassembled ribonucleoprotein (RNP) complex between a Cas9 nuclease and an sgRNA. This nanoparticle has an addition of a RVG peptide YTIWMPENPRPGTPCDIFTNSRGKRASNG which specifically interacts withthe N-acetylecholine receptor (AchR) on neuronal cells, which mediates NP entry Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
AB_141607 - Antibody [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: A21202 Invitrogen, Donkey anti-mouse alexafluor 488 Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
CleanCap® Cas9 mRNA (5moU) - Genome Editor [Small Animal Testing Center (SATC)] Matched on: --> Ecto-epithelium Epithelium SpCas9 mRNA with 2 NLS signals, HA tag and capped using CleanCap. It is polyadenylated, substituted with a modified uridine and optimized for mammalian systems. It mimics a fully processed mature mRNA. Experiments Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear
sNLS-SpCas9-sNLS - Genome Editor [Small Animal Testing Center (SATC), Delivery Systems Initiative] Matched on: --> Ecto-epithelium Epithelium SpCas9 with N- and C-terminal SV40 NLS Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
RNP-NC-CPP - Delivery System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium The nanocapsule is a thin glutathione (GSH)-cleavable covalently crosslinked polymer coating around a preassembled ribonucleoprotein (RNP) complex between a Cas9 nuclease and an sgRNA. This nanoparticle has an addition of a cell penetrating peptide (CPP) from the TAT peptide (GRKKRRQRRRPQ) which lacks cell-type specficity Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
L2-modified - Guide [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo, In Vitro] Matched on: --> Ecto-epithelium Epithelium This gRNA targets the Ai9 and related transgenes Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
BI28:AAV-GFAP-SaCas9-WPRE3-pA - Vector [Small Animal Testing Center (SATC), Delivery Systems Initiative] Matched on: --> Ecto-epithelium Epithelium Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
AB_2286684 - Antibody [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: sc-17320 Sox-2 (Y-17) Antibody, Santa Cruz Biotechnology Experiments Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium Asokan Aravind A dual vector strategy was employed: one delivering a single guide RNA and CB driven SaCas9, and another delivering the second guide RNA and CB driven SaCas9. This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 2e12vg was injected into each mouse (1e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
AAV9_pTR_self comp 2xU6-Ai9 guides - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
AAVcc47-Ai9-sgRNA1-CB-SaCas9 - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
AAV9-CMV-Cre - Vector [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium AAV serotype 9 delivering CMV Cre Recombinase Experiments Cre Recombinase dose escalation study in Ai9 mice
AAV9-Ai9-sgRNA2-CB-SaCas9 - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
AAVcc47-CMV-SaCas9 - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAVcc47 delivering CMV driven SaCas9 Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
sgAi9L - Guide Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium This sgRNA targets the Ai9 and related transgenes
SpyCas9-3xNLS - Genome Editor [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Epithelium SpyCas9-3xNLS is type II-A Cas9 from Streptococcus pyogenes strain SF370. It was expressed from pMCSG7 bacterial expressing vector and purified from Escherichia coli Rosetta DE3 strain. SpyCas9 fused to 3 NLS: C-Myc-like NLS at the N-terminal SV40 NLS and Nucleoplasmin NLS at the C-terminal Experiments Testing newly chemically modified crRNA and tracrRNA in mTmG mouse embryonic fibroblasts Testing newly chemically modified crRNA and tracrRNA to activate the TLR1 reporter in human cells Testing newly chemically modified crRNA and tracrRNA in mouse Hepa 1-6 cells Testing newly chemically modified crRNA and tracrRNA in mouse Neuro 2A cells Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center Testing newly chemically modified crRNA and tracrRNA to activate the TLR reporter in human cells
C57BL/6J mouse - Model System [Delivery Systems Initiative] - [In Vivo] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Foregut epithelium C57BL/6J WT mouse Experiments Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice
AAVcc47-CMV-Cre - Vector [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium AAVcc47 delivering CMV Cre Recombinase Experiments Cre Recombinase dose escalation study in Ai9 mice
Ai14 mouse - Model System [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Ai14 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. The att site flanked neo selection cassette has been removed in this strain. Experiments Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
306-O12B - Delivery System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo, In Vitro] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Unilaminar epithelium Combinatorial library cationic lipid nanoparticles Experiments Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Asokan Aravind C57/BL6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding a GFP reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
AAV9-GFP - Vector [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium AAV2/9 self complementary vector expressing GFP driven by CBh promoter Experiments Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
C57BL/6 mouse (Asokan study) - Model System [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Experiments Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
AB_2813835 - Antibody [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: ab150155 Abcam, Donkey anti-rat alexa fluour 647 Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
RRID:AB_10711040 - Antibody Matched on: --> Ecto-epithelium Epithelium Other Id: Abcam, ab104224 Mouse monoclonal [1B7] to NeuN - Neuronal Marker
AAVcc47-SaCas9-Ai9 - Vector [Small Animal Testing Center (SATC)] Matched on: --> Columnar epithelium Foregut epithelium Epithelium Endo-epithelium Meso-epithelium AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
[Validation] Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear - Experiment [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Murray Stephen A Delivery of CRISPR/Cas9 editor via bioreducible lipid nanoparticle to the inner ear in Ai14 mice Projects The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi)
sg298 - Guide [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium This sgRNA targets the Ai9 and related transgenes at multiple sites. 2'-O-Methyl at 3 first and last bases, 3' phosphorothioate bonds between first 3 and last 2 bases Experiments Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear
[Validation] Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain - Experiment [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Heaney Jason D Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing. Editing detected and quantified in brain by generation of tdTomato fluorescent protein signal from Ai9 reporter mice Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin)
R2-modified - Guide [Delivery Systems Initiative] - [In Vivo, In Vitro] Matched on: --> Ecto-epithelium Epithelium This gRNA targets the Ai9 and related transgenes Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AB_10015251 - Antibody [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: 25-6790 Anti-Myosin VIIa antibody, Proteus Biosciences Experiments Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
RRID:AB_2762834 - Antibody Matched on: --> Ecto-epithelium Epithelium Other Id: ThermoFisher #A32794 Alexa fluor 555, Donkey anti-rabbit
RRID:AB_141607 - Antibody Matched on: --> Ecto-epithelium Epithelium Other Id: Thermofisher #A21202 Alexa fluor 488, Donkey anti-mouse
RNP-NC-no ligand - Delivery System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium The nanocapsule is a thin glutathione (GSH)-cleavable covalently crosslinked polymer coating around a preassembled ribonucleoprotein (RNP) complex between a Cas9 nuclease and an sgRNA. Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
Ai14 gRNA - Guide [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium This sgRNA targets the Ai9 and related transgenes at multiple sites Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
RRID:AB_10563941 - Antibody Matched on: --> Ecto-epithelium Epithelium Other Id: Invitrogen, R10367 Polyclonal rabbit anti-RFP antibody
Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Digestive tract epithelium Columnar epithelium Epithelium Unilaminar epithelium Endo-epithelium Chen Zheng-Yi Delivery of firefly luciferase mRNA via new Lipid NanoParticles by tail vein injection into WT C57BL/6J mice targeting the Liver and delivery is measured by luciferase expression. Projects Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Deverman Benjamin E 3e11 vg/mouse of AAV-BI28:GFAP-SaCas9-WPRE-pA and 3e11 vg/mouse of AAV-BI28:GFAP-NLS-GFP-U6-L1-U6-R2 were codelivered intravenously to adult male and female Ai9 mice. Editing was assessed in brain sections 4 weeks later. Projects Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2 - Vector [Small Animal Testing Center (SATC), Delivery Systems Initiative] Matched on: --> Ecto-epithelium Epithelium Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
L1-modified - Guide [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo, In Vitro] Matched on: --> Ecto-epithelium Epithelium This gRNA targets the Ai9 and related transgenes Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
AAVcc47-Cre - Vector [Small Animal Testing Center (SATC)] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAV2/5 expressing Cre recombinase Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Cre Recombinase dose escalation study in Ai9 mice - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Asokan Aravind A single stranded cmv cre cassette was packaged into AAV9 or AAVcc47 and injected intravenously in Ai9 mice. We injected n=3 at three different doses (1e10, 1e11, 1e12 vg) and harvested organs 4 weeks post injection. Fluorescence intensity in liver, heart, and skeletal muscle was quantified with tiff based images in Image J and neuronal transduction from each vector was quantified at the 1e12vg dose by counting the number of tdTomato+ neurons and number of NeuN+ cells from multiple sections and images. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Chen Zheng-Yi Delivery of Cas9/sgRNA mRNA via new LNPs to the cochlea by cochleostomy and gene editing is measured by percentage of tdTomato positive cells. Projects Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles
AAV9-mCherry - Vector [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter Experiments Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
AAVcc47-mCherry - Vector [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter Experiments Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
AAVcc84-GFP - Vector [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter Experiments Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Sontheimer Erik J Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Projects Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors
[Validation] Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain - Experiment [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Murray Stephen A Delivery of CRISPR/Cas9 via RNP-loaded nanocages to the brain in Ai14 mice Projects The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin)
AB_2532994 - Antibody [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: 13-0300 Thermo-Fisher, Rat anti-GFAP Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
AB_141637 - Antibody [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: A21207 Invitrogen, Donkey anti-rabbit alexa fluor 594 Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
RRID:AB_2633281 - Antibody Matched on: --> Ecto-epithelium Epithelium Other Id: Invitrogen Product #A32732 Goat anti-rabbit IgG-Alexa Fluor 555
CleanCap® Fluc - Genome Editor [Delivery Systems Initiative] Matched on: --> Digestive tract epithelium Columnar epithelium Epithelium Unilaminar epithelium Endo-epithelium Firefly luciferase mRNA capped using CleanCap. It is polyadenylated, modified with 5-methoxyuridine and optimized for mammalian systems. It mimics a fully processed mature mRNA. Experiments Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice
306-O12B blank - Delivery System [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Combinatorial library cationic lipid nanoparticles Experiments Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium Asokan Aravind A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=3) and AAVcc47 (n=3) by intravenous injection in Ai9 mice. A total dose of 3e12vg was injected into each mouse (1.5e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium Asokan Aravind A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:4 ratio of cas9 to guide RNA (1e12vg of CMV Sacas9 vector and 3e12vg of the sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
AAV9-Ai9-sgRNA1-CB-SaCas9 - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
AAV9-CMV-SaCas9 - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAV serotype 9 delivering CMV driven SaCas9 Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
AAVcc47-Ai9-sgRNA1 + sgRNA2 - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
AAVcc47-Ai9-sgRNA2-CB-SaCas9 - Vector [Delivery Systems Initiative] Matched on: --> Columnar epithelium Epithelium Endo-epithelium Meso-epithelium Unilaminar epithelium AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
SaCas9 - Genome Editor [Small Animal Testing Center (SATC), Delivery Systems Initiative] Matched on: --> Ecto-epithelium Epithelium Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
AAVcc81-GFP - Vector [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Meso-epithelium AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter Experiments Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
306-S10 - Delivery System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo, In Vitro] Matched on: --> Ecto-epithelium Columnar epithelium Epithelium Endo-epithelium Unilaminar epithelium combinatorial library cationic lipid nanoparticles Experiments Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
Ai9 SaCas9 Guide A - Guide [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Columnar epithelium Foregut epithelium Epithelium Endo-epithelium Meso-epithelium This gRNA targets the Ai9 and related transgenes Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
CleanCap® Cas9 - Genome Editor [Delivery Systems Initiative] Matched on: --> Ecto-epithelium Epithelium SpCas9 mRNA with 2 NLS signals, HA tag and capped using CleanCap. It is polyadenylated, substituted with a modified uridine and optimized for mammalian systems. It mimics a fully processed mature mRNA. Experiments Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
STS204 (DNMT1; Sp_t2:Sp_c20_Dnmt1) - Guide [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium For endogenous locus Experiments Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center
RRID:AB_1196615 - Antibody [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: AB_1196615 GFP (D5.1) XP Rabbit mAb antibody, Cell Signaling Technology Experiments Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA using the S10 shuttle peptide to activate the mTmG reporter in mouse brain Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain. - Experiment [Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Gong Shaoqin (Sarah) Nanocapusules carrying CRISPR Cas9 RNP with guide RNA targeting the stop sequence in the Ai14 transgene are intracerebrally delivered to Ai14 mice and gene editing is measured by gain of tdTomato protein expression. Projects Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain
AB_2209751 - Antibody [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: Rockland Cat# 600-401-379 Anti-RFP (RABBIT) Antibody Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
AB_10711040 - Antibody [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Other Id: ab104224 Abcam, mouse anti-NeuN Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
Matched on: --> Ecto-epithelium Epithelium Other Id: GeneTex Product # GTX00837 Chicken Polyclonal anti-NeuN antibody
RRID:AB_2762843 - Antibody Matched on: --> Ecto-epithelium Epithelium Other Id: Invitrogen Product # A32931 Goat anti-chicken IgG-Alexa Fluor 488
[Validation] Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear - Experiment [Small Animal Testing Center (SATC)] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Murray Stephen A Delivery of CRISPR/Cas9 via bioreducible lipid nanoparticles (LNPs) to the inner ear in Ai14 mice Projects The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi)
sg298 - Guide [Delivery Systems Initiative] - [In Vivo, In Vitro] Matched on: --> Ecto-epithelium Epithelium This sgRNA targets the Ai9 and related transgenes at multiple sites Experiments Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
Ai14 mouse (congenic) - Model System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo] Matched on: --> Ecto-epithelium Epithelium Ai14 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. The att site flanked neo selection cassette has been removed in this strain. Experiments Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea

125 results for Epithelium

Category	Type	Name	Description	View Associated..
Experiment	In Vivo	Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.	Delivery of GFP via shuttle peptides to mouse airway epithelium via nasal instilation. Delivery efficiency was quantified in large and small airways by counting the number of GFP positive cells divided by the number of DAPI cells.	Projects Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Genome Editor	Cas Nuclease	Alt-R® S.p. Cas9 Nuclease V3		Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
Guide		g-loxP2_C9	This sgRNA targets the Ai9 and related transgenes	Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
Model System	Animal	BALB/c mouse	BALB/cJ is a commonly used inbred. Key traits include a susceptibility to developing the demyelinating disease upon infection with Theiler's murine encephalomyelitis virus. The BALB/cJ substrain is susceptible to Listeria, all species of Leishmania, and several species of Trypanosoma, but is resistant to experimental allergic orchitis (EAO).	Experiments Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
Antibody			Anti-CC10 (Rabbit) Polyclonal Antibody, dilution used 1:2,000
Genome Editor	Cas Nuclease	AsCas12a (IDT and Feldan Therapeutics)		Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
Experiment	In Vivo	Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia	In vivo shuttle peptide delivery of Cas9 and Cas12a RNPs in mouse airway epithelia. Gene editing was quantified by the GFP+ cells in large and small airways following 1 delivery of GFP protein by GFP positive cells compared to DAPI stained cells.	Projects Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Genome Editor	Reporter	GFP-NLS	Nuclear targeted GFP	Experiments Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
Guide		sgAi9L	This sgRNA targets the Ai9 and related transgenes
Guide		sgAi9R	This sgRNA targets the Ai9 and related transgenes
Model System	Animal	mTmG mouse	ROSAmT/mG is a cell membrane-targeted, two-color fluorescent Cre-reporter allele. Prior to Cre recombination, cell membrane-localized tdTomato (mT) fluorescence expression is widespread in cells/tissues. Cre recombinase expressing cells (and future cell lineages derived from these cells) have cell membrane-localized EGFP (mG) fluorescence expression replacing the red fluorescence	Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
Genome Editor	Recombinase	Cre recombinase	Cre recombinase delivered by AAV (see vector details)	Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Vector	Viral Vector	ssAAV5-sgB.saCas9		Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Guide		BCM_ssAAV5-Sa_sgB	This gRNA targets the Ai9 and related transgenes	Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Antibody			Anti-RFP (RABBIT) Antibody
Antibody			Anti-RFP (Mouse) Monoclonal Antibody, dilution used 1:300
Antibody			Anti-RFP (Rabbit) Polyclonal Antibody
Antibody			Anti-alpha Tubulin (Mouse) Monoclonal Antibody, dilution used 1:200
Genome Editor	Cas Nuclease	SpCas9	HA-SV40NLS-SpCas9-SV40NLS	Experiments A novel human T cell platform to define biological adverse effects of genome editing
Vector	Viral Vector	AAV.pU1a-SpCas9	Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
Vector	Viral Vector	H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)	AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP
Guide		g-loxPbot_C12a	This sgRNA targets the Ai9 and related transgenes at two sites	Experiments Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
Genome Editor	Cas Nuclease	SaCas9		Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Delivery System	Amphiphilic peptide	S10	Shuttle peptide used to deliver reagents to airway epithelia	Experiments Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia. Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia Peptide Shuttle optimization to deliver Cas9 RNP to human airway epithelia cells Gene editing in vitro by various peptide variants delivering Cas RNPs to primary Human airway epithelia cells. Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA using the S10 shuttle peptide to activate the mTmG reporter in mouse brain Peptide Shuttle optimization to deliver Cas12a RNP to human airway epithelia cells Gene editing in vitro by various peptide variants delivering Cas12a in NK cells.
Vector	Viral Vector	scAAV5-Cre-GFP		Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Antibody			GFP (D5.1) XP Rabbit mAb antibody
Delivery System	Amphiphilic peptide	D10		Experiments Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Model System	Animal	Ai9 mouse	Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.	Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Cre Recombinase dose escalation study in Ai9 mice Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Delivery System	Amphiphilic peptide	D237		Experiments Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Genome Editor	Cas Nuclease	SpCas9		Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Guide		BCM_ssAAV5-Sp_sgB	This sgRNA targets the Ai9 and related transgenes	Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Guide		SpyCas9 g-loxP2_C9	This sgRNA targets the mTmG transgene	Experiments Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Vector	Viral Vector	ssAAV5-sgA+sgB-U1A.GFP		Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Model System	Animal	Ai9 mouse (BCM)	Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.	Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Model System	Animal	Ai9-SauSpyCas9 mouse	Ai9-SauSpyCas9 is a version of Ai9 that has a single guide RNA sites on both sides of the STOP cassette. This is a Cre reporter allele that has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Ai9 mice express robust tdTomato fluorescence following Cre-mediated recombination.	Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Experiment	In Vivo	Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides	Ribonucleoproteins for CRISPR/Cas9 editing are complexed with amphiphilic peptides for delivery to lung airway epithilia via intranasal instillation into mTmG reporter mice. Editing is detected by production of GFP protein, and green fluorescence in airway linings	Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - McCray Validation (McCray Jr., Paul)
Vector	Viral Vector	ssAAV5-spCas9		Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Guide		BCM_ssAAV5-Sp_sgA	This sgRNA targets the Ai9 and related transgenes	Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Model System	Animal	mTmG mouse (congenic)	mTmG is a double-fluorescent reporter transgenic mouse which expresses membrane-targeted tdTomato flanked by loxP sequences, followed by membrane-targeted GFP. After genomic cleavage by Cas9 at two sites, or Cre recombinase between loxP sites, tdTomato expression is lost and GFP is expressed.	Experiments Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA using the S10 shuttle peptide to activate the mTmG reporter in mouse brain Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain
Experiment	In Vivo	Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice	AAV5 encoding CRISPR/Cas editing machinery were delivered to the lungs of reporter mice by intratracheal instillation. After 4 weeks incubation, the mice were dissected and the lungs imaged for the presence of tdTomato fluorescence, indicating successful editing. Editing calculated by dividing the number of tdTomato+ red cells by the number of nuclei in each airway	Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Gao Validation (Gao, Guang-Ping )
Delivery System	Viral vector	AAV	See vector details	Experiments Cre Recombinase dose escalation study in Ai9 mice
Experiment	In Vivo	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.	A dual vector strategy was employed: one self complementary vector delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (single stranded vector). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=4) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:1 ratio of cas9 to guide RNA (2e12vg of CMV Sacas9 vector and 2e12vg of the self complementary sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Genome Editor	Cas Nuclease	SaCas9-Lagor		Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Vector	Viral Vector	AAV9-Ai9-sgRNA1 + sgRNA2	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus	Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Vector	Viral Vector	AAVcc47_pTR_self comp 2xU6-Ai9 guides	AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene	Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Delivery System	Nanoparticle	113-O12B	combinatorial library cationic lipid nanoparticles	Experiments Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
Experiment	In Vivo	Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.	C57/BL6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding an mCherry reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification.	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Experiment	In Vivo	Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.	Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues.	Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Asokan Validation (Asokan, Aravind)
Genome Editor	Cas Nuclease	SauCas9		Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Guide		Ai9 SaCas9 Guide B	This gRNA targets the Ai9 and related transgenes	Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Guide		STS159 (mTmG; Sp_t2:Sp_c20_mTmG)	Targets mTmG and related reporter transgenes	Experiments Testing newly chemically modified crRNA and tracrRNA in mTmG mouse embryonic fibroblasts Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center
Delivery System	Nanoparticle	RNP-NC-RVG	The nanocapsule is a thin glutathione (GSH)-cleavable covalently crosslinked polymer coating around a preassembled ribonucleoprotein (RNP) complex between a Cas9 nuclease and an sgRNA. This nanoparticle has an addition of a RVG peptide YTIWMPENPRPGTPCDIFTNSRGKRASNG which specifically interacts withthe N-acetylecholine receptor (AchR) on neuronal cells, which mediates NP entry	Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
Antibody		AB_141607	Invitrogen, Donkey anti-mouse alexafluor 488	Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
Genome Editor	Cas Nuclease	CleanCap® Cas9 mRNA (5moU)	SpCas9 mRNA with 2 NLS signals, HA tag and capped using CleanCap. It is polyadenylated, substituted with a modified uridine and optimized for mammalian systems. It mimics a fully processed mature mRNA.	Experiments Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear
Genome Editor	Cas Nuclease	sNLS-SpCas9-sNLS	SpCas9 with N- and C-terminal SV40 NLS	Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
Delivery System	Nanoparticle	RNP-NC-CPP	The nanocapsule is a thin glutathione (GSH)-cleavable covalently crosslinked polymer coating around a preassembled ribonucleoprotein (RNP) complex between a Cas9 nuclease and an sgRNA. This nanoparticle has an addition of a cell penetrating peptide (CPP) from the TAT peptide (GRKKRRQRRRPQ) which lacks cell-type specficity	Experiments Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
Guide		L2-modified	This gRNA targets the Ai9 and related transgenes	Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Vector	Viral Vector	BI28:AAV-GFAP-SaCas9-WPRE3-pA	Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter	Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Antibody		AB_2286684	Sox-2 (Y-17) Antibody, Santa Cruz Biotechnology	Experiments Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
Experiment	In Vivo	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)	A dual vector strategy was employed: one delivering a single guide RNA and CB driven SaCas9, and another delivering the second guide RNA and CB driven SaCas9. This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 2e12vg was injected into each mouse (1e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Vector	Viral Vector	AAV9_pTR_self comp 2xU6-Ai9 guides	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene	Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Vector	Viral Vector	AAVcc47-Ai9-sgRNA1-CB-SaCas9	AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus	Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Vector	Viral Vector	AAV9-CMV-Cre	AAV serotype 9 delivering CMV Cre Recombinase	Experiments Cre Recombinase dose escalation study in Ai9 mice
Vector	Viral Vector	AAV9-Ai9-sgRNA2-CB-SaCas9	AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus	Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Vector	Viral Vector	AAVcc47-CMV-SaCas9	AAVcc47 delivering CMV driven SaCas9	Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Guide		sgAi9L	This sgRNA targets the Ai9 and related transgenes
Genome Editor	Cas Nuclease	SpyCas9-3xNLS	SpyCas9-3xNLS is type II-A Cas9 from Streptococcus pyogenes strain SF370. It was expressed from pMCSG7 bacterial expressing vector and purified from Escherichia coli Rosetta DE3 strain. SpyCas9 fused to 3 NLS: C-Myc-like NLS at the N-terminal SV40 NLS and Nucleoplasmin NLS at the C-terminal	Experiments Testing newly chemically modified crRNA and tracrRNA in mTmG mouse embryonic fibroblasts Testing newly chemically modified crRNA and tracrRNA to activate the TLR1 reporter in human cells Testing newly chemically modified crRNA and tracrRNA in mouse Hepa 1-6 cells Testing newly chemically modified crRNA and tracrRNA in mouse Neuro 2A cells Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center Testing newly chemically modified crRNA and tracrRNA to activate the TLR reporter in human cells
Model System	Animal	C57BL/6J mouse	C57BL/6J WT mouse	Experiments Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice
Vector	Viral Vector	AAVcc47-CMV-Cre	AAVcc47 delivering CMV Cre Recombinase	Experiments Cre Recombinase dose escalation study in Ai9 mice
Model System	Animal	Ai14 mouse	Ai14 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. The att site flanked neo selection cassette has been removed in this strain.	Experiments Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
Delivery System	Nanoparticle	306-O12B	Combinatorial library cationic lipid nanoparticles	Experiments Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea

Contact SCGE

125 results for Epithelium

125 results for Epithelium

Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia. - Experiment [Delivery Systems Initiative] - [In Vivo]

Alt-R® S.p. Cas9 Nuclease V3 - Genome Editor [Delivery Systems Initiative]

g-loxP2_C9 - Guide [Delivery Systems Initiative] - [In Vivo]

BALB/c mouse - Model System [Delivery Systems Initiative] - [In Vivo]

AsCas12a (IDT and Feldan Therapeutics) - Genome Editor [Delivery Systems Initiative]

Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia - Experiment [Delivery Systems Initiative] - [In Vivo]

GFP-NLS - Genome Editor [Delivery Systems Initiative]

sgAi9L - Guide

sgAi9R - Guide

mTmG mouse - Model System [Delivery Systems Initiative] - [In Vivo]

Cre recombinase - Genome Editor [Small Animal Testing Center (SATC), Delivery Systems Initiative]

ssAAV5-sgB.saCas9 - Vector [Small Animal Testing Center (SATC)]

BCM_ssAAV5-Sa_sgB - Guide [Small Animal Testing Center (SATC)] - [In Vivo]

SpCas9 - Genome Editor [Biological Systems]

AAV.pU1a-SpCas9 - Vector

H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) - Vector

g-loxPbot_C12a - Guide [Delivery Systems Initiative] - [In Vivo]

SaCas9 - Genome Editor [Small Animal Testing Center (SATC)]

S10 - Delivery System [Delivery Systems Initiative] - [In Vivo, In Vitro]

scAAV5-Cre-GFP - Vector [Small Animal Testing Center (SATC)]

D10 - Delivery System [Small Animal Testing Center (SATC)] - [In Vivo]

Ai9 mouse - Model System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo]

D237 - Delivery System [Small Animal Testing Center (SATC)] - [In Vivo]

SpCas9 - Genome Editor [Small Animal Testing Center (SATC)]

BCM_ssAAV5-Sp_sgB - Guide [Small Animal Testing Center (SATC)] - [In Vivo]

SpyCas9 g-loxP2_C9 - Guide [Small Animal Testing Center (SATC)] - [In Vivo]

ssAAV5-sgA+sgB-U1A.GFP - Vector [Small Animal Testing Center (SATC)]

Ai9 mouse (BCM) - Model System [Small Animal Testing Center (SATC)] - [In Vivo]

Ai9-SauSpyCas9 mouse - Model System [Small Animal Testing Center (SATC)] - [In Vivo]

[Validation] Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides - Experiment [Small Animal Testing Center (SATC)] - [In Vivo]

ssAAV5-spCas9 - Vector [Small Animal Testing Center (SATC)]

BCM_ssAAV5-Sp_sgA - Guide [Small Animal Testing Center (SATC)] - [In Vivo]

mTmG mouse (congenic) - Model System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo]

[Validation] Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice - Experiment [Small Animal Testing Center (SATC)] - [In Vivo]

AAV - Delivery System [Delivery Systems Initiative] - [In Vivo]

Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector. - Experiment [Delivery Systems Initiative] - [In Vivo]

SaCas9-Lagor - Genome Editor [Delivery Systems Initiative]

AAV9-Ai9-sgRNA1 + sgRNA2 - Vector [Delivery Systems Initiative]

AAVcc47_pTR_self comp 2xU6-Ai9 guides - Vector [Delivery Systems Initiative]

113-O12B - Delivery System [Delivery Systems Initiative] - [In Vivo]

Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. - Experiment [Delivery Systems Initiative] - [In Vivo]

[Validation] Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. - Experiment [Small Animal Testing Center (SATC)] - [In Vivo]

SauCas9 - Genome Editor [Small Animal Testing Center (SATC)]

Ai9 SaCas9 Guide B - Guide [Small Animal Testing Center (SATC)] - [In Vivo]

STS159 (mTmG; Sp_t2:Sp_c20_mTmG) - Guide [Delivery Systems Initiative] - [In Vivo, In Vitro]

RNP-NC-RVG - Delivery System [Small Animal Testing Center (SATC)] - [In Vivo]

AB_141607 - Antibody [Small Animal Testing Center (SATC)] - [In Vivo]

CleanCap® Cas9 mRNA (5moU) - Genome Editor [Small Animal Testing Center (SATC)]

sNLS-SpCas9-sNLS - Genome Editor [Small Animal Testing Center (SATC), Delivery Systems Initiative]

RNP-NC-CPP - Delivery System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo]

L2-modified - Guide [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo, In Vitro]

BI28:AAV-GFAP-SaCas9-WPRE3-pA - Vector [Small Animal Testing Center (SATC), Delivery Systems Initiative]

AB_2286684 - Antibody [Delivery Systems Initiative] - [In Vivo]

Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) - Experiment [Delivery Systems Initiative] - [In Vivo]

AAV9_pTR_self comp 2xU6-Ai9 guides - Vector [Delivery Systems Initiative]

AAVcc47-Ai9-sgRNA1-CB-SaCas9 - Vector [Delivery Systems Initiative]

AAV9-CMV-Cre - Vector [Delivery Systems Initiative]

AAV9-Ai9-sgRNA2-CB-SaCas9 - Vector [Delivery Systems Initiative]

AAVcc47-CMV-SaCas9 - Vector [Delivery Systems Initiative]

sgAi9L - Guide

SpyCas9-3xNLS - Genome Editor [Delivery Systems Initiative]

C57BL/6J mouse - Model System [Delivery Systems Initiative] - [In Vivo]

AAVcc47-CMV-Cre - Vector [Delivery Systems Initiative]

Ai14 mouse - Model System [Delivery Systems Initiative] - [In Vivo]

306-O12B - Delivery System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo, In Vitro]

Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. - Experiment [Delivery Systems Initiative] - [In Vivo]

AAV9-GFP - Vector [Delivery Systems Initiative]

C57BL/6 mouse (Asokan study) - Model System [Delivery Systems Initiative] - [In Vivo]

AB_2813835 - Antibody [Small Animal Testing Center (SATC)] - [In Vivo]

RRID:AB_10711040 - Antibody

AAVcc47-SaCas9-Ai9 - Vector [Small Animal Testing Center (SATC)]

[Validation] Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear - Experiment [Small Animal Testing Center (SATC)] - [In Vivo]

sg298 - Guide [Small Animal Testing Center (SATC)] - [In Vivo]

[Validation] Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain - Experiment [Small Animal Testing Center (SATC)] - [In Vivo]

R2-modified - Guide [Delivery Systems Initiative] - [In Vivo, In Vitro]

AB_10015251 - Antibody [Delivery Systems Initiative] - [In Vivo]

RRID:AB_2762834 - Antibody