178 results for Epithelium
178 results for Epithelium
Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia- [In Vivo] [Delivery Systems Initiative] [Mouse] |
GFP-NLS- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1)
Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
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SpCas9- [In Vivo, In Vitro] [Delivery Systems Initiative, Biological Systems] [Human, Mouse] |
g-loxP2_C9- [In Vivo] [Delivery Systems Initiative] [Mouse] |
g-loxPbot_C12a- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AsCas12a (IDT and Feldan Therapeutics)- [In Vivo] [Delivery Systems Initiative] [Mouse] |
- [In Vivo] [Delivery Systems Initiative] [Mouse]Anti-RFP (Mouse) Monoclonal Antibody, dilution used 1:300 Show Experiments (1)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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Alt-R® S.p. Cas9 Nuclease V3- [In Vivo] [Delivery Systems Initiative] [Mouse] |
sgAi9L- [In Vivo, In Vitro] [Delivery Systems Initiative] [Human, Mouse] |
sgAi9R- [In Vivo, In Vitro] [Delivery Systems Initiative] [Human, Mouse] |
Cre recombinase- [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]Show Experiments (3)
Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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- [In Vivo] [Delivery Systems Initiative] [Mouse]Anti-alpha Tubulin (Mouse) Monoclonal Antibody, dilution used 1:200 Show Experiments (1)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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BCM_ssAAV5-Sa_sgB- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Testing AAV5 for activation of tdTomato in mouse airway- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Other Id:
RRID:AB_310759
07-623
rabbit polyclonal antibody against club cell secretory protein, unconjugated, 07-623, EMD Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
|
Other Id:
905501.0
RRID:AB_2565050
rabbit polyclonal antibody to detect cytokeratin5+ basal cells, uncojugated, 905501, Biolegend Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
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BCM_ssAAV5-Sp_sgB- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
[Validation] Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
S10- [In Vivo, In Vitro] [Collaborative Opportunity Fund, Delivery Systems Initiative] [Human, Mouse, Rhesus macaque]Show Experiments (12)
Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
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ssAAV5-sgA+sgB-U1A.GFP- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
SpyCas9 g-loxP2_C9- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
mTmG mouse (congenic)- [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]Show Experiments (6)
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
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Ai9-SauSpyCas9 mouse- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Macaca mulatta (Rhesus Macaque)- [In Vivo] [Collaborative Opportunity Fund] [Rhesus macaque]Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
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- [In Vivo] [Delivery Systems Initiative] [Mouse]Anti-RFP (RABBIT) Antibody Show Experiments (1) |
scAAV5-Cre-GFP- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)- [In Vivo] [Delivery Systems Initiative] [Mouse] |
- [In Vivo] [Delivery Systems Initiative] [Mouse]Anti-RFP (Rabbit) Polyclonal Antibody Show Experiments (1)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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Other Id:
RRID:AB_477585
T6793
mouse monocolonal antibody against the acetylated α-tubulin, unconjugated, T6793, Sigma-Aldrich Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
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Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway- [In Vivo] [Collaborative Opportunity Fund] [Rhesus macaque]Show Project |
[Validation] Independent validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
SpCas9- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
D237- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
ssAAV5-spCas9- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Heaney_SATC Tissue Processing, Imaging and Analysis- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]Show Experiments (4)
Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
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SaCas9-Lagor- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (4)
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
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AAVcc47-Ai9-sgRNA1-CB-SaCas9- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Ai9 mouse- [In Vivo] [Small Animal Testing Center (SATC), AAV tropism, Delivery Systems Initiative] [Mouse]Show Experiments (10)
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
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TadA-8e V106W- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Cre Recombinase dose escalation study in Ai9 mice- [In Vivo] [Delivery Systems Initiative] [Mouse] |
sNLS-SpCas9-sNLS- [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.- [In Vivo] [Delivery Systems Initiative] [Mouse] |
SauCas9- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Ai9 SaCas9 Guide B- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
gRNA #8- [In Vivo, In Vitro] [Collaborative Opportunity Fund] [Rhesus macaque] |
On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Project |
CleanCap® Fluc- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1)
Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice
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AAV-Pcsk9- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Chaikof-Associated Protocol 3_Serum ELISAs- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1)
Pcsk9 adenine base editor efficiency in liver and nonliver tissue
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AAVcc47-Cre- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
C57BL/6J mouse- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (3)
Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice
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AAVcc47-CMV-Cre- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1) |
AAV4-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
CleanCap® Cas9 mRNA (5moU)- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
RNP-NC-CPP- [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
Ai14 gRNA- [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV9-GFP- [In Vivo] [Delivery Systems Initiative] [Mouse] |
[Validation] Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Ai9 SaCas9 Guide A- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Testing preparation for independent validation at The Jackson Laboratory Small Animal Testing Center- [In Vivo] [Delivery Systems Initiative] [Mouse] |
STS159 (mTmG; Sp_t2:Sp_c20_mTmG)- [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] |
STS204 (DNMT1; Sp_t2:Sp_c20_Dnmt1)- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Ai14 mouse- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Murray-SATC_Gong-Validaiton_Gong Study Protocol- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Other Id:
Rockland Cat# 600-401-379
AB_2209751
Anti-RFP (RABBIT) Antibody |
[Validation] Repeat experiment of independent validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Other Id:
AB_10015251
25-6790
Anti-Myosin VIIa antibody, Proteus Biosciences |
[Validation] Independent validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Other Id:
AB_141637
A21207
Invitrogen, Donkey anti-rabbit alexa fluor 594 |
null [Mouse]Chicken Polyclonal anti-NeuN antibody |
- [In Vivo] [Delivery Systems Initiative] [Mouse]GFP (D5.1) XP Rabbit mAb antibody Show Experiments (1) |
BALB/c mouse- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1)
Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
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mTmG mouse- [In Vivo] [Delivery Systems Initiative] [Mouse] |
SaCas9- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
ssAAV5-sgB.saCas9- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
AAV.pU1a-SpCas9- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells- [In Vivo] [Delivery Systems Initiative] [Mouse] |
- [In Vivo] [Delivery Systems Initiative] [Mouse]Anti-CC10 (Rabbit) Polyclonal Antibody, dilution used 1:2,000 Show Experiments (1)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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Other Id:
RRID:AB_2717534
PA5-71680
rabbit polyclonal antibody against the pulmonary-associated surfactant protein C (SPC), unconjugated, PA5-71680, Invitrogen Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
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Deverman_Comprehensive Methods- [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] |
D10- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Ai9 mouse (BCM)- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Heaney-SATC_McCray-Validation_Lung Inflation and Fixation Protocol- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Heaney-SATC_McCray-Validation_Intranasal Instillation in Mice Protocol- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
BCM_ssAAV5-Sp_sgA- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
BI28:AAV-GFAP-SaCas9-WPRE3-pA- [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Chaikof-Associated Protocol 2_Off-target editing AND Primers for sequencing analysis- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV9-CMV-SaCas9- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (3)
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)
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AAVcc47-Ai9-sgRNA2-CB-SaCas9- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAVcc47_pTR_self comp 2xU6-Ai9 guides- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Pcsk9 Exon 1 splice domain- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1)
Pcsk9 adenine base editor efficiency in liver and nonliver tissue
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AAV3b-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
AAV9-CMV-Cre- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1) |
RNP-NC-no ligand- [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
RNP-NC-RVG- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
113-O12B- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV9-mCherry- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAVcc81-GFP- [In Vivo] [Delivery Systems Initiative] [Mouse] |
CleanCap® Cas9- [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] |
306-S10- [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]Show Experiments (4)
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea
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AAVcc47-SaCas9-Ai9- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
AAV9-Ai9-sgRNA1-CB-SaCas9- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV9-Ai9-sgRNA1 + sgRNA2- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV9-Ai9-sgRNA2-CB-SaCas9- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAVcc47-Ai9-sgRNA1 + sgRNA2- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV5-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
AAV7-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
AAV9-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
AAVrh10-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
AAVrh74-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
AAVrh8-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
Murray-SATC_Gong-Validation Brain Injection in Mice Protocol- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Other Id:
AB_2532994
13-0300Â
Thermo-Fisher, Rat anti-GFAP |
Other Id:
ab150155
AB_2813835
Abcam, Donkey anti-rat alexa fluour 647 |
Other Id:
AB_141607
A21202
Invitrogen, Donkey anti-mouse alexafluor 488 |
306-O12B- [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]Show Experiments (5)
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea
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AAVcc84-GFP- [In Vivo] [Delivery Systems Initiative] [Mouse] |
C57BL/6 mouse (Asokan study)- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Ai14 mouse (congenic)- [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]Show Experiments (4)
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
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ABE8e-Cas9- [In Vivo, In Vitro] [Collaborative Opportunity Fund] [Human, Rhesus macaque]Show Experiments (3)
Delivery of ABE8e-Cas9 RNP using Shuttle peptide candidates to human airway epithelial cells cultured at the air liquid interface targeting B2M locus
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FSD315- [In Vivo, In Vitro] [Collaborative Opportunity Fund, Delivery Systems Initiative] [Human, Rhesus macaque]Show Experiments (7)
Peptide Shuttle optimization to deliver Cas9 RNP to human airway epithelia cells
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L2-modified- [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
SaCas9- [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]Show Experiments (3)
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
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L1-modified- [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]Show Experiments (3)
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
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Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)- [In Vivo] [Delivery Systems Initiative] [Mouse] |
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.- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV9_pTR_self comp 2xU6-Ai9 guides- [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAVcc47-CMV-SaCas9- [In Vivo] [Delivery Systems Initiative] [Mouse] |
ABE8e- [In Vitro] [Collaborative Opportunity Fund] [Mouse, Rhesus macaque]Show Experiments (1) |
Pcsk9 adenine base editor efficiency in liver and nonliver tissue- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Project |
Chaikof-Associated Protocol 1_Retro-orbital administration and High throughput sequencing- [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1)
Pcsk9 adenine base editor efficiency in liver and nonliver tissue
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Other Id:
AB_2286684
sc-17320
Sox-2 (Y-17) Antibody, Santa Cruz Biotechnology |
AAV Tropism project- [In Vivo] [AAV tropism] [Mouse] |
AAV- [In Vivo, In Vitro] [Biological Effects Initiative, AAV tropism, Delivery Systems Initiative] [Human, Mouse]Show Experiments (9)
Cre Recombinase dose escalation study in Ai9 mice
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AAV6-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
AAV8-ZsGreen-Cre- [In Vivo] [AAV tropism] [Mouse]Show Experiments (1) |
sg298- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
AAVcc47-mCherry- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Other Id:
RRID:AB_2536526
AB_2536526
GFP Recombinant Rabbit Monoclonal Antibody, Thermo Fisher Scientific #G10362 Show Experiments (2)
Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain
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Gong_Intracranial Injection Procedure for Mice- [In Vivo] [Delivery Systems Initiative] [Mouse] |
Other Id:
AB_10711040
ab104224Â
Abcam, mouse anti-NeuN |
306-O12B blank- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
[Validation] Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.- [In Vivo] [Delivery Systems Initiative] [Mouse] |
R2-modified- [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] |
Deverman_Area Based Quantification of Editing Efficiency Protocol- [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] |
BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2- [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
SpyCas9-3xNLS- [In Vivo, In Vitro] [Delivery Systems Initiative] [Human, Mouse]Show Experiments (6)
Testing newly chemically modified crRNA and tracrRNA to activate the TLR reporter in human cells
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Other Id:
AB_1196615
RRID:AB_1196615
GFP (D5.1) XP Rabbit mAb antibody, Cell Signaling Technology Show Experiments (3)
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
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Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.- [In Vivo] [Delivery Systems Initiative] [Mouse] |
[Validation] Independent validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear- [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea- [In Vivo] [Delivery Systems Initiative] [Mouse] |
sg298- [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] |
178 results for Epithelium
Category | Name | Description | Source | View Associated.. |
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Experiment | 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 | GFP-NLS | Nuclear targeted GFP | Expressed From (Escherichia coli BL21DE3) |
Show Experiments (1)
Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
|
Genome Editor | SpCas9 | HA-SV40NLS-SpCas9-SV40NLS | Vector Encoded | |
Guide | g-loxP2_C9 | This sgRNA targets the Ai9 and related transgenes | IDT | |
Guide | g-loxPbot_C12a | This sgRNA targets the Ai9 and related transgenes at two sites | IDT | |
Genome Editor | AsCas12a (IDT and Feldan Therapeutics) | IDT and Feldan Therapeutics | ||
Antibody | Anti-RFP (Mouse) Monoclonal Antibody, dilution used 1:300 |
Show Experiments (1)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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Genome Editor | Alt-R® S.p. Cas9 Nuclease V3 | Recombinant S. pyogenes Cas9 nuclease, purified from an E. coli strain expressing the nuclease. Contains nuclear localization sequence (NLS) and C-terminal 6-His tag. Provided in solution at 10 µg/µL. 100 µg of Cas9 nuclease = 610 pmol. | Integrated DNA Technologies | |
Guide | sgAi9L | This sgRNA targets the Ai9 and related transgenes | IDT | |
Guide | sgAi9R | This sgRNA targets the Ai9 and related transgenes | IDT | |
Genome Editor | Cre recombinase | Cre recombinase delivered by AAV (see vector details) |
Show Experiments (3)
Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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Antibody | Anti-alpha Tubulin (Mouse) Monoclonal Antibody, dilution used 1:200 |
Show Experiments (1)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
|
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Guide | BCM_ssAAV5-Sa_sgB | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Experiment | 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 | ||
Antibody | RRID:AB_310759 | rabbit polyclonal antibody against club cell secretory protein, unconjugated, 07-623, EMD |
Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
|
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Antibody | RRID:AB_2565050 | rabbit polyclonal antibody to detect cytokeratin5+ basal cells, uncojugated, 905501, Biolegend |
Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
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Guide | BCM_ssAAV5-Sp_sgB | This sgRNA targets the Ai9 and related transgenes | Vector encoded | |
Experiment | [Validation] Independent 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 | ||
Protocol | Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice | Procedure for Intratracheal (IT) delivery of AAV in mouse lung. | ||
Delivery System | S10 | Shuttle peptide used to deliver reagents to airway epithelia | McCray Lab |
Show Experiments (12)
Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
|
Vector | ssAAV5-sgA+sgB-U1A.GFP | Gao Lab | ||
Guide | SpyCas9 g-loxP2_C9 | This sgRNA targets the mTmG transgene | IDT | |
Model System | 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. | The Jackson Laboratory |
Show Experiments (6)
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
|
Model System | 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. | Baylor College of Medicine | |
Experiment | 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 | Macaca mulatta (Rhesus Macaque) | Tarantal Lab |
Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
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Antibody | Anti-RFP (RABBIT) Antibody |
Show Experiments (1) |
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Vector | scAAV5-Cre-GFP | Gao Lab | ||
Vector | H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) | AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP | Addgene Gao Lab | |
Antibody | Anti-RFP (Rabbit) Polyclonal Antibody |
Show Experiments (1)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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Antibody | RRID:AB_477585 | mouse monocolonal antibody against the acetylated α-tubulin, unconjugated, T6793, Sigma-Aldrich |
Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
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Experiment | Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway | We utilized novel amphiphilic shuttle peptides to deliver base editor ribonucleoprotein (RNP) into the airways to edit airway epithelial cells (CCR5 locus) of rhesus monkeys. The Cas9-ABE8e RNP and shuttle peptides S10 or FSD315 were aerosolized into the rhesus monkey trachea. Seven days later, tissues were obtained and dissected, and airway epithelia collected from the trachea, mainstem, and segmental bronchi using cytology brushes. DNA was extracted from epithelial cells and subjected to high-throughput sequencing. Using the FSD315 shuttle peptide and Cas9-ABE8e, we achieved a mean editing efficiency of 2.8% at the CCR5 locus in airway epithelial cells (range 0.02 – 5.3%) depending on the anatomic region sampled. To visualize the biodistribution of the RNPs within the respiratory tract and in specific cell types, we delivered a Cy5-fluorescent peptide fused to a nuclear localization signal (NLS-Cy5) using the S10 peptide. The lungs were obtained 1 and 2 hours post-delivery, fixed, and examined by microscopy. Epifluorescence and confocal microscopy documented an effective intra-nuclear delivery of NLS-Cy5 into epithelial cells throughout the respiratory tract, including large, medium, and small airways, and alveolar regions. Ongoing analyses will identify the NLS-Cy5-positive epithelial cell types using co-localization with fluorescently-labeled antibodies. In summary, using a rhesus monkey model, following a single delivery of adenine base editor RNPs to the airways in a clinically relevant manner we achieved up to 5.3% editing efficiency of the CCR5 locus in airway epithelia, a level considered therapeutically relevant in cystic fibrosis. |
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Experiment | [Validation] Independent 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 | ||
Genome Editor | SpCas9 | IDT | ||
Delivery System | D237 | McCray Lab | ||
Vector | ssAAV5-spCas9 | Gao Lab | ||
Protocol | Heaney_SATC Tissue Processing, Imaging and Analysis | Procedure for tissue preparation, imaging and analysis. |
Show Experiments (4)
Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
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Genome Editor | SaCas9-Lagor | William Lagor, Baylor College Of Medicine |
Show Experiments (4)
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
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Vector | AAVcc47-Ai9-sgRNA1-CB-SaCas9 | AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus | Asokan Lab | |
Model System | 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. | The Jackson Laboratory |
Show Experiments (10)
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
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Genome Editor | TadA-8e V106W | Catalytically impaired Cas fused to evolved TadA deaminase (TadA-8e V106W) | Chaikof Lab | |
Experiment | 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. | ||
Vector | demo VV04-Cre | DEMO viral vector for demo purpose | Virus Company X | |
Vector | demo VV05-Cre | DEMO viral vector for demo purpose | Virus Company X | |
Genome Editor | sNLS-SpCas9-sNLS | SpCas9 with N- and C-terminal SV40 NLS | Aldevron 9212-5MG | |
Antibody | RRID:AB_2813835 | |||
Experiment | Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. | C57BL/6 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. | ||
Genome Editor | SauCas9 | |||
Guide | Ai9 SaCas9 Guide B | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | gRNA #8 | IDT | ||
Experiment | On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver | On-target editing compared to off-target editing at 14 CIRCLE seq nominated sites in livers of an adenine base editor delivered by engineered virus-like particles (BE-eVLPs). Treated mice vs. untreated vs. AAV was assessed one week after systemic administration of BE-eVLPs or AAV-Pcsk9 to C57BL/6 mice. DNA sequencing reads containing A-T to G-C mutations within protospacer positions 4-10. |
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Genome Editor | 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. | Trilink Biotechnologies |
Show Experiments (1)
Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice
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Vector | AAV-Pcsk9 | |||
Experiment | 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. | ||
Protocol | Chaikof-Associated Protocol 3_Serum ELISAs | This protocol describes in vivo adminstration and subsequent blood draws and ELISA on C57BL/6J mice. |
Show Experiments (1)
Pcsk9 adenine base editor efficiency in liver and nonliver tissue
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Vector | AAVcc47-Cre | AAV2/5 expressing Cre recombinase | Asokan Lab | |
Model System | C57BL/6J mouse | C57BL/6J WT mouse | The Jackson Laboratory |
Show Experiments (3)
Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice
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Vector | AAVcc47-CMV-Cre | AAVcc47 delivering CMV Cre Recombinase | Asokan Lab |
Show Experiments (1) |
Vector | AAV4-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Genome Editor | 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. | Trilink Biotechnologies | |
Delivery System | 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 | Gong Lab | |
Guide | Ai14 gRNA | This sgRNA targets the Ai9 and related transgenes at multiple sites | IDT | |
Antibody | RRID:AB_10711040 | |||
Experiment | Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. | C57BL/6 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. | ||
Vector | AAV9-GFP | AAV2/9 self complementary vector expressing GFP driven by CBh promoter | Asokan Lab | |
Experiment | [Validation] Independent 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. | ||
Guide | Ai9 SaCas9 Guide A | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Experiment | Testing preparation for independent 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 | ||
Guide | STS159 (mTmG; Sp_t2:Sp_c20_mTmG) | Targets mTmG and related reporter transgenes | In house production | |
Guide | STS204 (DNMT1; Sp_t2:Sp_c20_Dnmt1) | For endogenous locus | In house production | |
Antibody | RRID:AB_2313606 | Vector Labs Goat Anti-Rabbit IgG Biotinylated Cat. #BA-1000 | ||
Model System | 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. | The Jackson Laboratory | |
Protocol | Murray-SATC_Gong-Validaiton_Gong Study Protocol | Procedure for intracranial injection, immunofluorescence and imaging. | ||
Antibody | AB_2209751 | Anti-RFP (RABBIT) Antibody | ||
Antibody | RRID:AB_2762843 | Goat anti-chicken IgG-Alexa Fluor 488 | ||
Experiment | [Validation] Repeat experiment of independent 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 | ||
Antibody | RRID:AB_10563941 | Polyclonal rabbit anti-RFP antibody | ||
Antibody | RRID:AB_2762834 | Alexa fluor 555, Donkey anti-rabbit | ||
Antibody | AB_10015251 | Anti-Myosin VIIa antibody, Proteus Biosciences | ||
Experiment | [Validation] Independent 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_141637 | Invitrogen, Donkey anti-rabbit alexa fluor 594 | ||
Antibody | Chicken Polyclonal anti-NeuN antibody | |||
Antibody | GFP (D5.1) XP Rabbit mAb antibody |
Show Experiments (1) |
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Model System | 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). | The Jackson Laboratory |
Show Experiments (1)
Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
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Model System | mTmG mouse | ROSAmT/mGÂ 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 | The Jackson Laboratory | |
Genome Editor | SaCas9 | Vector Encoded (ssaav5-sga+sgb-u1a.gfp) | ||
Vector | ssAAV5-sgB.saCas9 | Gao Lab | ||
Vector | AAV.pU1a-SpCas9 | Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS | Addgene Gao Lab | |
Experiment | Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells | 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. Gene editing quantified by tdTomato activation and cell specific markers for club and ciliated cell types. | ||
Antibody | Anti-CC10 (Rabbit) Polyclonal Antibody, dilution used 1:2,000 |
Show Experiments (1)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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Antibody | RRID:AB_2717534 | rabbit polyclonal antibody against the pulmonary-associated surfactant protein C (SPC), unconjugated, PA5-71680, Invitrogen |
Show Experiments (1)
Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
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Protocol | Deverman_Comprehensive Methods | Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue processing and IHC. | ||
Delivery System | D10 | McCray Lab | ||
Model System | 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. | Baylor College of Medicine | |
Protocol | Heaney-SATC_McCray-Validation_Lung Inflation and Fixation Protocol | Procedure for mouse lung inflation and fixation. | ||
Protocol | Heaney-SATC_McCray-Validation_Intranasal Instillation in Mice Protocol | Procedure for intranasal instillation of ribonucleoprotein/peptide complex in mice for delivery to the lung. | ||
Guide | BCM_ssAAV5-Sp_sgA | This sgRNA targets the Ai9 and related transgenes | Vector encoded | |
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 | Deverman Lab | |
Experiment | 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. | ||
Protocol | Chaikof-Associated Protocol 2_Off-target editing AND Primers for sequencing analysis | This protocol describes in vivo adminstration and subsequent analysis of off-target editing in the liver. | ||
Experiment | 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 | AAV9-CMV-SaCas9 | AAV serotype 9 delivering CMV driven SaCas9 | Asokan Lab |
Show Experiments (3)
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)
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Vector | AAVcc47-Ai9-sgRNA2-CB-SaCas9 | AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus | Asokan Lab | |
Vector | AAVcc47_pTR_self comp 2xU6-Ai9 guides | AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene | Asokan Lab | |
Guide | Pcsk9 Exon 1 splice domain | Pcsk9 Exon 1 splice domain | Synthego |
Show Experiments (1)
Pcsk9 adenine base editor efficiency in liver and nonliver tissue
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Vector | AAV3b-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | AAV9-CMV-Cre | AAV serotype 9 delivering CMV Cre Recombinase | Asokan Lab |
Show Experiments (1) |
Delivery System | 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. | Gong Lab | |
Delivery System | 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 | Gong Lab | |
Antibody | RRID:AB_141607 | |||
Delivery System | 113-O12B | combinatorial library cationic lipid nanoparticles | Xu lab | |
Vector | AAV9-mCherry | AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter | Asokan Lab | |
Vector | AAVcc81-GFP | AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter | Asokan Lab | |
Genome Editor | 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. | Trilink Biotechnologies | |
Delivery System | 306-S10 | combinatorial library cationic lipid nanoparticles | Xu lab |
Show Experiments (4)
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea
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Vector | AAVcc47-SaCas9-Ai9 | AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter | Asokan Lab | |
Vector | AAV9-Ai9-sgRNA1-CB-SaCas9 | AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus | Asokan Lab | |
Vector | AAV9-Ai9-sgRNA1 + sgRNA2 | AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus | Asokan Lab | |
Vector | AAV9-Ai9-sgRNA2-CB-SaCas9 | AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus | Asokan Lab | |
Vector | AAVcc47-Ai9-sgRNA1 + sgRNA2 | AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus | Asokan Lab | |
Antibody | RRID:AB_141607 | Alexa fluor 488, Donkey anti-mouse | ||
Vector | AAV5-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | AAV7-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | AAV9-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | AAVrh10-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | AAVrh74-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | AAVrh8-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | demo VV02-Cre | DEMO viral vector for demo purpose | Virus Company X | |
Vector | demo VV03-Cre | DEMO viral vector for demo purpose | Virus Company X | |
Antibody | RRID:AB_2209751 | |||
Antibody | RRID:AB_2532994 | |||
Protocol | Murray-SATC_Gong-Validation Brain Injection in Mice Protocol | Procedure for brain injection surgical procedure, pre- and post-operative care for mice. | ||
Antibody | AB_2532994 | Thermo-Fisher, Rat anti-GFAP | ||
Antibody | AB_2813835 | Abcam, Donkey anti-rat alexa fluour 647 | ||
Antibody | AB_141607 | Invitrogen, Donkey anti-mouse alexafluor 488 | ||
Antibody | RRID:AB_2633281 | Goat anti-rabbit IgG-Alexa Fluor 555 | ||
Delivery System | 306-O12B | Combinatorial library cationic lipid nanoparticles | Xu lab |
Show Experiments (5)
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea
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Vector | AAVcc84-GFP | AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter | Asokan Lab | |
Model System | C57BL/6 mouse (Asokan study) | Unspecified | ||
Model System | 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. | The Jackson Laboratory |
Show Experiments (4)
Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea
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Genome Editor | ABE8e-Cas9 | A-to-G base editor | Feldan Therapeutics |
Show Experiments (3)
Delivery of ABE8e-Cas9 RNP using Shuttle peptide candidates to human airway epithelial cells cultured at the air liquid interface targeting B2M locus
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Delivery System | FSD315 | Shuttle peptide used to deliver reagents to airway epithelia | McCray Lab |
Show Experiments (7)
Peptide Shuttle optimization to deliver Cas9 RNP to human airway epithelia cells
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Guide | L2-modified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Genome Editor | SaCas9 |
Show Experiments (3)
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
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Guide | L1-modified | This gRNA targets the Ai9 and related transgenes | Vector encoded |
Show Experiments (3)
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
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Experiment | 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. | ||
Experiment | 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 | AAV9_pTR_self comp 2xU6-Ai9 guides | AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene | Asokan Lab | |
Vector | AAVcc47-CMV-SaCas9 | AAVcc47 delivering CMV driven SaCas9 | Asokan Lab | |
Genome Editor | ABE8e | Tad8e deaminase fused to a nickase (D10A) spCas9 | David Liu Lab |
Show Experiments (1) |
Experiment | Pcsk9 adenine base editor efficiency in liver and nonliver tissue | Adenine base editing at the Pcsk9 exon 1 splice donor site in mouse heart, kidney, liver, lungs, muscle, and spleen was assessed one week after systemic administration of an adenine base editor delivered by engineered virus-like particles (BE-eVLPs) in C56BL/6 mice |
Show Project |
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Protocol | Chaikof-Associated Protocol 1_Retro-orbital administration and High throughput sequencing | This Protocol describes in vivo administration of BE-eVLP for Pcsk9 knockdown in liver, including sequencing endpoint. |
Show Experiments (1)
Pcsk9 adenine base editor efficiency in liver and nonliver tissue
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Antibody | AB_2286684 | Sox-2 (Y-17) Antibody, Santa Cruz Biotechnology | ||
Delivery System | eVLP | engineered virus like particles | Liu Lab | |
Experiment | AAV Tropism project | Ten AAV serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized for biodistribution across 20 tissues by quantitative PCR and imaging | ||
Delivery System | AAV | See vector details |
Show Experiments (9)
Cre Recombinase dose escalation study in Ai9 mice
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Vector | AAV6-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | AAV8-ZsGreen-Cre | AAV backbone with a bi-directional promoter driving zsGreen and Cre | Guangping Gao Lab |
Show Experiments (1) |
Vector | demo VV01-Cre | DEMO viral vector for demo purpose | Virus Company X | |
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 | Synthego | |
Antibody | RRID:AB_141637 | |||
Vector | AAVcc47-mCherry | AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter | Asokan Lab | |
Antibody | RRID:AB_2536526 | GFP Recombinant Rabbit Monoclonal Antibody, Thermo Fisher Scientific #G10362 |
Show Experiments (2)
Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain
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Protocol | Gong_Intracranial Injection Procedure for Mice | Procedure for intracranial delivery to mouse brain. | ||
Antibody | RRID:AB_10711040 | Mouse monoclonal [1B7] to NeuN - Neuronal Marker | ||
Antibody | AB_10711040 | Abcam, mouse anti-NeuN | ||
Delivery System | 306-O12B blank | Combinatorial library cationic lipid nanoparticles | Xu lab | |
Experiment | [Validation] Independent 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 | ||
Experiment | 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. | ||
Guide | R2-modified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Protocol | Deverman_Area Based Quantification of Editing Efficiency Protocol | Procedure for non-IHC based image quantification of editing. | ||
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 | Deverman Lab | |
Genome Editor | 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 | Sontheimer lab |
Show Experiments (6)
Testing newly chemically modified crRNA and tracrRNA to activate the TLR reporter in human cells
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Antibody | RRID:AB_1196615 | GFP (D5.1) XP Rabbit mAb antibody, Cell Signaling Technology |
Show Experiments (3)
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
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Experiment | 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. | ||
Experiment | [Validation] Independent 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 | 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. | ||
Guide | sg298 | This sgRNA targets the Ai9 and related transgenes at multiple sites | Synthego |