Search Result for Epithelium

List View
Table View

93 results for Epithelium

Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia. - Experiment - [In Vivo] Matched on: description -> Delivery of GFP via shuttle peptides to mouse airway epithelium via nasal instilation. generatedDescription -> Delivery of GFP via shuttle peptides to mouse airway epithelium via nasal instilation. tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> 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. View Associated:
BALB/c mouse - Model System - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> 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). View Associated:
Alt-R® S.p. Cas9 Nuclease V3 - Genome Editor Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> Lung epithelium ; Epithelium of mucosa ; Epithelium ; Foregut epithelium ; Endo-epithelium View Associated:
GFP-NLS - Genome Editor Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> Lung epithelium ; Epithelium of mucosa ; Epithelium ; Foregut epithelium ; Endo-epithelium Nuclear targeted GFP View Associated:
Testing AAV5 for activation of tdTomato in mouse airway - Experiment - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Lung epithelium Gao Guang-Ping AAV2/5 mediated gene editing in the mouse airway was tested by deliverying SpCas9 and guide RNAs targeting the Ai9 transgene in Ai9 transgenic mice. Viral delivery was detected by GFP expression and gene editing quantified by tdTomato activation View Associated:
Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia - Experiment - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> 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. View Associated:
AsCas12a (IDT and Feldan Therapeutics) - Genome Editor Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> Lung epithelium ; Epithelium of mucosa ; Epithelium ; Foregut epithelium ; Endo-epithelium View Associated:
g-loxP2_C9 - Guide - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> Lung epithelium ; Epithelium of mucosa ; Epithelium ; Foregut epithelium ; Endo-epithelium This sgRNA targets the Ai9 and related transgenes View Associated:
SpCas9 - Genome Editor Matched on: tissueTerm -> Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Lung epithelium HA-SV40NLS-SpCas9-SV40NLS View Associated: Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases A Novel Human T-Cell Platform to Define Biological Effects of Genome Editing Testing AAV5 for activation of tdTomato in HEK293T cells Testing AAV5 for activation of tdTomato in mouse airway A novel human T cell platform to define biological adverse effects of genome editing
AAV.pU1a-SpCas9 - Vector Matched on: tissueTerm -> Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Lung epithelium Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS View Associated:
H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) - Vector Matched on: tissueTerm -> Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Lung epithelium AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP View Associated:
g-loxPbot_C12a - Guide - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> Lung epithelium ; Epithelium of mucosa ; Epithelium ; Foregut epithelium ; Endo-epithelium This sgRNA targets the Ai9 and related transgenes at two sites View Associated:
mTmG mouse - Model System - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> 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 View Associated:
S10 - Delivery System - [In Vivo, In Vitro] Matched on: tissueTerm -> Epithelium of bronchiole ; Epithelium of main bronchus Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Lung epithelium Shuttle peptide used to deliver reagents to airway epithelia View Associated: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors 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.
AB_1196615 - Antibody - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Lung epithelium GFP (D5.1) XP Rabbit mAb antibody View Associated:
AB_2209751 - Antibody - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Ecto-epithelium ; Epithelium ; Endo-epithelium ; Lung epithelium Anti-RFP (RABBIT) Antibody View Associated: Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Testing AAV5 for activation of tdTomato in mouse airway
Ai9 mouse - Model System - [In Vivo] Matched on: tissueTerm -> Epithelium of bronchiole Tissue/CellType Term Synonyms -> 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. View Associated: Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System Evolving High Potency AAV Vectors for Neuromuscular Genome Editing BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) 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) Testing AAV5 for activation of tdTomato in mouse airway 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.
mTmG mouse (congenic) - Model System - [In Vivo] Matched on: tissueTerm -> Epithelium of main bronchus ; Epithelium of bronchiole Tissue/CellType Term Synonyms -> 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. View Associated: BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - McCray Validation (McCray Jr., Paul) Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors 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
SpCas9 - Genome Editor Matched on: tissueTerm -> Epithelium of main bronchus ; Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Lung epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium View Associated:
Ai9 mouse (BCM) - Model System - [In Vivo] Matched on: tissueTerm -> Epithelium of main bronchus ; Epithelium of bronchiole Tissue/CellType Term Synonyms -> 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. View Associated:
[] Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides - Experiment - [In Vivo] Matched on: tissueTerm -> Epithelium of main bronchus ; Epithelium of bronchiole Tissue/CellType Term Synonyms -> 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 View Associated:
D10 - Delivery System - [In Vivo] Matched on: tissueTerm -> Epithelium of main bronchus ; Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Lung epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium View Associated:
D237 - Delivery System - [In Vivo] Matched on: tissueTerm -> Epithelium of main bronchus ; Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Lung epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium View Associated:
SpyCas9 g-loxP2_C9 - Guide - [In Vivo] Matched on: tissueTerm -> Epithelium of main bronchus ; Epithelium of bronchiole Tissue/CellType Term Synonyms -> Epithelium of mucosa ; Lung epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium This sgRNA targets the mTmG transgene View Associated:
AAVcc47_pTR_self comp 2xU6-Ai9 guides - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene View Associated:
Cre Recombinase dose escalation study in Ai9 mice - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
306-O12B - Delivery System - [In Vivo, In Vitro] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Unilaminar epithelium Combinatorial library cationic lipid nanoparticles View Associated: Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi) 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 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
AAVcc84-GFP - Vector Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter View Associated:
[] Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
AAV9-Ai9-sgRNA1-CB-SaCas9 - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus View Associated:
AAV9-Ai9-sgRNA2-CB-SaCas9 - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus View Associated:
AAVcc47-Ai9-sgRNA1-CB-SaCas9 - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus View Associated:
AAVcc47-Ai9-sgRNA1 + sgRNA2 - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus View Associated: 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)
Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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 View Associated:
Cre recombinase - Genome Editor Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium Cre recombinase delivered by AAVcc47-Cre viral vector View Associated:
AAVcc47-CMV-Cre - Vector Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium AAVcc47 delivering CMV Cre Recombinase View Associated:
RNP-NC-CPP - Delivery System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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 View Associated: The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain 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.
CleanCap® Fluc - Genome Editor Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
SaCas9-Lagor - Genome Editor Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium View Associated: 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_pTR_self comp 2xU6-Ai9 guides - Vector Matched on: Tissue/CellType Term Synonyms -> 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 View Associated:
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
AAVcc47-Cre - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAV2/5 expressing Cre recombinase View Associated:
AAV9-CMV-Cre - Vector Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium AAV serotype 9 delivering CMV Cre Recombinase View Associated:
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
AAV9-GFP - Vector Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium AAV2/9 self complementary vector expressing GFP driven by CBh promoter View Associated:
AAVcc81-GFP - Vector Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter View Associated:
SauCas9 - Genome Editor Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Foregut epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium View Associated:
CleanCap® Cas9 - Genome Editor Matched on: Tissue/CellType Term Synonyms -> 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. View Associated: 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
sg298 - Guide - [In Vivo, In Vitro] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium This sgRNA targets the Ai9 and related transgenes at multiple sites View Associated: 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
AB_10015251 - Antibody - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Anti-Myosin VIIa antibody, Proteus Biosciences View Associated:
AB_1196615 - Antibody - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium GFP (D5.1) XP Rabbit mAb antibody, Cell Signaling Technology View Associated:
Ai14 gRNA - Guide - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium This sgRNA targets the Ai9 and related transgenes at multiple sites View Associated: The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain 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.
[] Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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 View Associated:
SaCas9 - Genome Editor Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium View Associated: Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) 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
Ai14 mouse - Model System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
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 - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
AAV9-Ai9-sgRNA1 + sgRNA2 - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus View Associated: 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 Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus View Associated:
AAVcc47-CMV-SaCas9 - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAVcc47 delivering CMV driven SaCas9 View Associated: 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)
AB_2286684 - Antibody - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Sox-2 (Y-17) Antibody, Santa Cruz Biotechnology View Associated:
CleanCap® Cas9 mRNA (5moU) - Genome Editor Matched on: Tissue/CellType Term Synonyms -> 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. View Associated: 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 Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium SpCas9 with N- and C-terminal SV40 NLS View Associated: The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain 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.
C57BL/6J mouse - Model System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Foregut epithelium C57BL/6J WT mouse View Associated:
113-O12B - Delivery System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Unilaminar epithelium combinatorial library cationic lipid nanoparticles View Associated: 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 - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
C57BL/6 mouse (Asokan study) - Model System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium View Associated: 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.
[] Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Murray Stephen A Delivery of CRISPR/Cas9 via RNP-loaded nanocages to the brain in Ai14 mice View Associated:
AB_2532994 - Antibody - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Thermo-Fisher, Rat anti-GFAP View Associated:
AB_141637 - Antibody - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Invitrogen, Donkey anti-rabbit alexa fluor 594 View Associated:
AB_141607 - Antibody - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Invitrogen, Donkey anti-mouse alexafluor 488 View Associated:
306-O12B blank - Delivery System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Combinatorial library cationic lipid nanoparticles View Associated:
BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2 - Vector Matched on: Tissue/CellType Term Synonyms -> 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 View Associated: Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) 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
Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
AAV9-CMV-SaCas9 - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium ; Unilaminar epithelium AAV serotype 9 delivering CMV driven SaCas9 View Associated: 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-mCherry - Vector Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter View Associated:
AAVcc47-mCherry - Vector Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter View Associated:
306-S10 - Delivery System - [In Vivo, In Vitro] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Columnar epithelium ; Epithelium ; Endo-epithelium ; Unilaminar epithelium combinatorial library cationic lipid nanoparticles View Associated: Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi) 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
AAVcc47-SaCas9-Ai9 - Vector Matched on: Tissue/CellType Term Synonyms -> Columnar epithelium ; Foregut epithelium ; Epithelium ; Endo-epithelium ; Meso-epithelium AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter View Associated:
Ai14 mouse (congenic) - Model System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated: Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi) 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
SpyCas9-3xNLS - Genome Editor Matched on: Tissue/CellType Term Synonyms -> 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 View Associated: Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors 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
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain. - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated:
RNP-NC-RVG - Delivery System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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 View Associated:
AB_10711040 - Antibody - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Abcam, mouse anti-NeuN View Associated:
AB_2813835 - Antibody - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Abcam, Donkey anti-rat alexa fluour 647 View Associated:
[] Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Murray Stephen A Delivery of CRISPR/Cas9 via bioreducible lipid nanoparticles (LNPs) to the inner ear in Ai14 mice View Associated:
[] Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear - Experiment - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Murray Stephen A Delivery of CRISPR/Cas9 editor via bioreducible lipid nanoparticle to the inner ear in Ai14 mice View Associated:
RNP-NC-no ligand - Delivery System - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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. View Associated: The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain 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.
sg298 - Guide - [In Vivo] Matched on: Tissue/CellType Term Synonyms -> 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 View Associated: 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
BI28:AAV-GFAP-SaCas9-WPRE3-pA - Vector Matched on: Tissue/CellType Term Synonyms -> Ecto-epithelium ; Epithelium Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter View Associated: Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) 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

93 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.
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).
Genome Editor	Cas Nuclease	Alt-R® S.p. Cas9 Nuclease V3
Genome Editor	Reporter	GFP-NLS	Nuclear targeted GFP
Experiment	In Vivo	Testing AAV5 for activation of tdTomato in mouse airway	AAV2/5 mediated gene editing in the mouse airway was tested by deliverying SpCas9 and guide RNAs targeting the Ai9 transgene in Ai9 transgenic mice. Viral delivery was detected by GFP expression and gene editing quantified by tdTomato activation
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.
Genome Editor	Cas Nuclease	AsCas12a (IDT and Feldan Therapeutics)
Guide		g-loxP2_C9	This sgRNA targets the Ai9 and related transgenes
Genome Editor	Cas Nuclease	SpCas9	HA-SV40NLS-SpCas9-SV40NLS	Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases A Novel Human T-Cell Platform to Define Biological Effects of Genome Editing Testing AAV5 for activation of tdTomato in HEK293T cells Testing AAV5 for activation of tdTomato in mouse airway 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
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
Delivery System	Amphiphilic peptide	S10	Shuttle peptide used to deliver reagents to airway epithelia	Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors 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.
Antibody		AB_1196615	GFP (D5.1) XP Rabbit mAb antibody
Antibody		AB_2209751	Anti-RFP (RABBIT) Antibody	Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Testing AAV5 for activation of tdTomato in mouse airway
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.	Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System Evolving High Potency AAV Vectors for Neuromuscular Genome Editing BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) 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) Testing AAV5 for activation of tdTomato in mouse airway 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.
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.	BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - McCray Validation (McCray Jr., Paul) Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors 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
Genome Editor	Cas Nuclease	SpCas9
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.
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
Delivery System	Amphiphilic peptide	D10
Delivery System	Amphiphilic peptide	D237
Guide		SpyCas9 g-loxP2_C9	This sgRNA targets the mTmG transgene
Vector	Viral Vector	AAVcc47_pTR_self comp 2xU6-Ai9 guides	AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene
Experiment	In Vivo	Cre Recombinase dose escalation study in Ai9 mice	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.
Delivery System	Nanoparticle	306-O12B	Combinatorial library cationic lipid nanoparticles	Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi) 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
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.
Vector	Viral Vector	AAVcc84-GFP	AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter
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.
Experiment	In Vivo	Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)	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.
Vector	Viral Vector	AAV9-Ai9-sgRNA1-CB-SaCas9	AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
Vector	Viral Vector	AAV9-Ai9-sgRNA2-CB-SaCas9	AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus
Vector	Viral Vector	AAVcc47-Ai9-sgRNA1-CB-SaCas9	AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
Vector	Viral Vector	AAVcc47-Ai9-sgRNA1 + sgRNA2	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus	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)
Experiment	In Vivo	Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center	Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain
Genome Editor	Recombinase	Cre recombinase	Cre recombinase delivered by AAVcc47-Cre viral vector
Vector	Viral Vector	AAVcc47-CMV-Cre	AAVcc47 delivering CMV Cre Recombinase
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	The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain 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.
Genome Editor	Reporter	CleanCap® Fluc	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.
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)	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.
Genome Editor	Cas Nuclease	SaCas9-Lagor		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_pTR_self comp 2xU6-Ai9 guides	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene
Experiment	In Vivo	Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.	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.
Vector	Viral Vector	AAVcc47-Cre	AAV2/5 expressing Cre recombinase
Vector	Viral Vector	AAV9-CMV-Cre	AAV serotype 9 delivering CMV Cre Recombinase
Experiment	In Vivo	Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea	Delivery of Cas9/sgRNA mRNA via new LNPs to the cochlea by cochleostomy and gene editing is measured by percentage of tdTomato positive cells.
Vector	Viral Vector	AAV9-GFP	AAV2/9 self complementary vector expressing GFP driven by CBh promoter
Vector	Viral Vector	AAVcc81-GFP	AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter
Genome Editor	Cas Nuclease	SauCas9
Genome Editor	Cas Nuclease	CleanCap® Cas9	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.	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
Guide		sg298	This sgRNA targets the Ai9 and related transgenes at multiple sites	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
Antibody		AB_10015251	Anti-Myosin VIIa antibody, Proteus Biosciences
Antibody		AB_1196615	GFP (D5.1) XP Rabbit mAb antibody, Cell Signaling Technology
Guide		Ai14 gRNA	This sgRNA targets the Ai9 and related transgenes at multiple sites	The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain 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.
Experiment	In Vivo	Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain	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
Genome Editor	Cas Nuclease	SaCas9		Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) 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
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.
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.
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.
Vector	Viral Vector	AAV9-Ai9-sgRNA1 + sgRNA2	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus	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-Ai9-sgRNA2-CB-SaCas9	AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus
Vector	Viral Vector	AAVcc47-CMV-SaCas9	AAVcc47 delivering CMV driven SaCas9	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)
Antibody		AB_2286684	Sox-2 (Y-17) Antibody, Santa Cruz Biotechnology
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.	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	The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain 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.
Model System	Animal	C57BL/6J mouse	C57BL/6J WT mouse
Delivery System	Nanoparticle	113-O12B	combinatorial library cationic lipid nanoparticles	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 8 (VR8) 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 a GFP reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification.
Model System	Animal	C57BL/6 mouse (Asokan study)		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.
Experiment	In Vivo	Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain	Delivery of CRISPR/Cas9 via RNP-loaded nanocages to the brain in Ai14 mice
Antibody		AB_2532994	Thermo-Fisher, Rat anti-GFAP
Antibody		AB_141637	Invitrogen, Donkey anti-rabbit alexa fluor 594
Antibody		AB_141607	Invitrogen, Donkey anti-mouse alexafluor 488
Delivery System	Nanoparticle	306-O12B blank	Combinatorial library cationic lipid nanoparticles
Vector	Viral Vector	BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2	Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds	Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) 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
Experiment	In Vivo	Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice	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.
Vector	Viral Vector	AAV9-CMV-SaCas9	AAV serotype 9 delivering CMV driven SaCas9	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-mCherry	AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter
Vector	Viral Vector	AAVcc47-mCherry	AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter
Delivery System	Nanoparticle	306-S10	combinatorial library cationic lipid nanoparticles	Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi) 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
Vector	Viral Vector	AAVcc47-SaCas9-Ai9	AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter
Model System	Animal	Ai14 mouse (congenic)	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.	Efficient In Vivo RNP-Based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) The Jackson Laboratory Gene Editing Testing Center - Chen Validation (Chen, Zheng-Yi) 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
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	Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors 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
Experiment	In Vivo	Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.	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.
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
Antibody		AB_10711040	Abcam, mouse anti-NeuN
Antibody		AB_2813835	Abcam, Donkey anti-rat alexa fluour 647
Experiment	In Vivo	Second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear	Delivery of CRISPR/Cas9 via bioreducible lipid nanoparticles (LNPs) to the inner ear in Ai14 mice
Experiment	In Vivo	Repeat experiment of second site validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear	Delivery of CRISPR/Cas9 editor via bioreducible lipid nanoparticle to the inner ear in Ai14 mice
Delivery System	Nanoparticle	RNP-NC-no ligand	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.	The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Enabling Nanoplatforms for Targeted In Vivo Delivery of CRISPR/Cas9 Riboncleoproteins in the Brain 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		sg298	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	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
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	Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) 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

Contact SCGE

93 results for Epithelium

93 results for Epithelium

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

BALB/c mouse - Model System - [In Vivo]

Alt-R® S.p. Cas9 Nuclease V3 - Genome Editor

GFP-NLS - Genome Editor

Testing AAV5 for activation of tdTomato in mouse airway - Experiment - [In Vivo]

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

AsCas12a (IDT and Feldan Therapeutics) - Genome Editor

g-loxP2_C9 - Guide - [In Vivo]

SpCas9 - Genome Editor

AAV.pU1a-SpCas9 - Vector

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

g-loxPbot_C12a - Guide - [In Vivo]

mTmG mouse - Model System - [In Vivo]

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

AB_1196615 - Antibody - [In Vivo]

AB_2209751 - Antibody - [In Vivo]

Ai9 mouse - Model System - [In Vivo]

mTmG mouse (congenic) - Model System - [In Vivo]

SpCas9 - Genome Editor

Ai9 mouse (BCM) - Model System - [In Vivo]

[] Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides - Experiment - [In Vivo]

D10 - Delivery System - [In Vivo]

D237 - Delivery System - [In Vivo]

SpyCas9 g-loxP2_C9 - Guide - [In Vivo]

AAVcc47_pTR_self comp 2xU6-Ai9 guides - Vector

Cre Recombinase dose escalation study in Ai9 mice - Experiment - [In Vivo]

306-O12B - Delivery System - [In Vivo, In Vitro]

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

AAVcc84-GFP - Vector

[] Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. - Experiment - [In Vivo]

Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) - Experiment - [In Vivo]

AAV9-Ai9-sgRNA1-CB-SaCas9 - Vector

AAV9-Ai9-sgRNA2-CB-SaCas9 - Vector

AAVcc47-Ai9-sgRNA1-CB-SaCas9 - Vector

AAVcc47-Ai9-sgRNA1 + sgRNA2 - Vector

Testing preparation for validation at The Jackson Laboratory Small Animal Testing Center - Experiment - [In Vivo]

Cre recombinase - Genome Editor

AAVcc47-CMV-Cre - Vector

RNP-NC-CPP - Delivery System - [In Vivo]

CleanCap® Fluc - Genome Editor

Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) - Experiment - [In Vivo]

SaCas9-Lagor - Genome Editor

AAV9_pTR_self comp 2xU6-Ai9 guides - Vector

Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. - Experiment - [In Vivo]

AAVcc47-Cre - Vector

AAV9-CMV-Cre - Vector

Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea - Experiment - [In Vivo]

AAV9-GFP - Vector

AAVcc81-GFP - Vector

SauCas9 - Genome Editor

CleanCap® Cas9 - Genome Editor

sg298 - Guide - [In Vivo, In Vitro]

AB_10015251 - Antibody - [In Vivo]

AB_1196615 - Antibody - [In Vivo]

Ai14 gRNA - Guide - [In Vivo]

[] Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain - Experiment - [In Vivo]

SaCas9 - Genome Editor

Ai14 mouse - Model System - [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 - [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 - [In Vivo]

AAV9-Ai9-sgRNA1 + sgRNA2 - Vector

AAVcc47-Ai9-sgRNA2-CB-SaCas9 - Vector

AAVcc47-CMV-SaCas9 - Vector

AB_2286684 - Antibody - [In Vivo]

CleanCap® Cas9 mRNA (5moU) - Genome Editor

sNLS-SpCas9-sNLS - Genome Editor

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

113-O12B - Delivery System - [In Vivo]

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

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

[] Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain - Experiment - [In Vivo]

AB_2532994 - Antibody - [In Vivo]

AB_141637 - Antibody - [In Vivo]

AB_141607 - Antibody - [In Vivo]

306-O12B blank - Delivery System - [In Vivo]

BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2 - Vector

Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice - Experiment - [In Vivo]