86 results for Ai9
86 results for Ai9
Ai9-SauSpyCas9 mouseModel System - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Ai9 mouse (BCM)Model System - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Ai9 mouseModel System - [In Vivo] [Small Animal Testing Center (SATC), AAV tropism, Delivery Systems Initiative] [Mouse]Show Experiments (11)
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
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Ai9 mouse immortalized fibroblastsModel System - [In Vitro] [Delivery Systems Initiative] [Mouse] |
Ai9 SaCas9 Guide BGuide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
HEK-293T with Ai9 transient reporter assayModel System - [In Vitro] [Delivery Systems Initiative] [Human]Show Experiments (1) |
Ai9 SaCas9 Guide AGuide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Cre Recombinase dose escalation study in Ai9 miceExperiment - [In Vivo] [Delivery Systems Initiative] [Mouse] |
FUS (focused ultrasound) array validation in Ai9 miceExperiment - [In Vivo] [Delivery Systems Initiative] [Mouse] |
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] |
Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitroExperiment - [In Vitro] [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)Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] |
[Validation] Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 miceExperiment - [In Vivo] [Small Animal Testing Center (SATC)] [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.Experiment - [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)Experiment - [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)Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] |
Ai9LR21-SaCas9Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1) |
AAV+Focused UltrasoundDelivery System - [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1) |
SaCas9Genome Editor - [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1) |
Sa_Ai9_LGuide - [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1) |
Sa_Ai9_RGuide - [In Vivo] [Delivery Systems Initiative] [Mouse]Show Experiments (1) |
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] |
RNP-NC-RVGDelivery System - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Other Id:
AB_141637
A21207
Invitrogen, Donkey anti-rabbit alexa fluor 594 |
Other Id:
AB_141607
A21202
Invitrogen, Donkey anti-mouse alexafluor 488 |
AAVcc47-SaCas9-Ai9Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Gong_Intracranial Injection Procedure for MiceProtocol - [In Vivo] [Delivery Systems Initiative] [Mouse] |
Other Id:
ab150155
AB_2813835
Abcam, Donkey anti-rat alexa fluour 647 |
Murray-SATC_Gong-Validation Brain Injection in Mice ProtocolProtocol - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Other Id:
Rockland Cat# 600-401-379
AB_2209751
Anti-RFP (RABBIT) Antibody |
R2-modifiedGuide - [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] |
sgAi9LGuide - [In Vivo, In Vitro] [Delivery Systems Initiative] [Human, Mouse] |
[Validation] Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.Experiment - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
SauCas9Genome Editor - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
[Validation] Independent validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brainExperiment - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Ai14 gRNAGuide - [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
Ai14 mouseModel System - [In Vivo] [Delivery Systems Initiative] [Mouse] |
Murray-SATC_Gong-Validaiton_Gong Study ProtocolProtocol - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
Other Id:
AB_2532994
13-0300Â
Thermo-Fisher, Rat anti-GFAP |
Other Id:
AB_10711040
ab104224Â
Abcam, mouse anti-NeuN |
Ai14 mouse (congenic)Model System - [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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BCM_ssAAV5-Sp_sgAGuide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
BCM_ssAAV5-Sp_sgBGuide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
L1-modifiedGuide - [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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L3-unmodifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
g-loxPbot_C12aGuide - [In Vivo] [Delivery Systems Initiative] [Mouse] |
sg298Guide - [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] |
L1-unmodifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
L3-modifedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
R1-unmodifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
RNP-NC-CPPDelivery System - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Collaborative Opportunity Fund, Delivery Systems Initiative] [Human, Mouse]Show Experiments (5)
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
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sNLS-SpCas9-sNLSGenome Editor - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Collaborative Opportunity Fund, Delivery Systems Initiative] [Human, Mouse]Show Experiments (5)
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
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g-loxP2_C9Guide - [In Vivo] [Delivery Systems Initiative] [Mouse] |
L2-modifiedGuide - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] |
L2-unmodifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
R2-unmodifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
sgAi9RGuide - [In Vivo, In Vitro] [Delivery Systems Initiative] [Human, Mouse] |
BCM_ssAAV5-Sa_sgBGuide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
R1-modifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
AAV9-Ai9-sgRNA1-CB-SaCas9Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAVcc47-Ai9-sgRNA2-CB-SaCas9Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] |
RNP-NC-no ligandDelivery System - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Collaborative Opportunity Fund, Delivery Systems Initiative] [Human, Mouse]Show Experiments (3)
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
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SaLoxP2-unmodifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
Testing AAV5 for activation of tdTomato in mouse airwayExperiment - [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAVcc47-Ai9-sgRNA1-CB-SaCas9Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] |
SaLoxP1-unmodifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
SaLoxP2-modifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
SaLoxP1-modifiedGuide - [In Vitro] [Delivery Systems Initiative] [Mouse] |
AAV9-Ai9-sgRNA2-CB-SaCas9Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV9-Ai9-sgRNA1 + sgRNA2Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAVcc47-Ai9-sgRNA1 + sgRNA2Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAVcc47_pTR_self comp 2xU6-Ai9 guidesVector - [In Vivo] [Delivery Systems Initiative] [Mouse] |
Heaney_SATC Tissue Processing, Imaging and AnalysisProtocol - [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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Testing AAV5 for activation of tdTomato in HEK293T cellsExperiment - [In Vitro] [Delivery Systems Initiative] [Human] |
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cellsExperiment - [In Vivo] [Delivery Systems Initiative] [Mouse] |
AAV Tropism projectExperiment - [In Vivo] [AAV tropism] [Mouse] |
AAV9_pTR_self comp 2xU6-Ai9 guidesVector - [In Vivo] [Delivery Systems Initiative] [Mouse] |
sg298Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
[Validation] Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brainExperiment - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] |
86 results for Ai9
Category | Name | Description | Source | View Associated.. |
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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 | |
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 | |
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 (11)
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
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Model System | Ai9 mouse immortalized fibroblasts | Immortalized fibroblasts made from Ai9 (B6.Cg-Gt(ROSA)26Sor^tm9(CAG-tdTomato)Hze/J) mice | ||
Guide | Ai9 SaCas9 Guide B | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Model System | HEK-293T with Ai9 transient reporter assay | HEK-293T cells transfected with an Ai9 inducible transgene reporter plasmid used to test gene editing activity by fluorescence. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient. |
Show Experiments (1) |
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Guide | Ai9 SaCas9 Guide A | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
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. | ||
Experiment | FUS (focused ultrasound) array validation in Ai9 mice | 9.3 week-old Ai9 mice (4 male and 4 female) were administered Ai9-targeting SaCas9 AAV9 vector through intravenous adminsitration (2E12 vg/mouse) and left hemisphere was targeted by FUS (focused ultrasound) array for BBB (blood brain barrier) opening | ||
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. | ||
Experiment | Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro | Reporter transgene activation by SaCas9 gRNA target and modified scaffold sequences by transient transfection in immortalized Ai9 mouse fibroblasts | ||
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 | [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 | ||
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. | ||
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. | ||
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 | Ai9LR21-SaCas9 | AAV9 encoding S. aureus Cas9 and two guide RNAs with modified scaffolds | Leong Lab |
Show Experiments (1) |
Delivery System | AAV+Focused Ultrasound | See vector details | Leong Lab |
Show Experiments (1) |
Genome Editor | SaCas9 | Leong Lab |
Show Experiments (1) |
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Guide | Sa_Ai9_L | This gRNA targets the Ai9 and related transgenes, has modified scaffold (Tabebordbar Science 2016) |
Show Experiments (1) |
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Guide | Sa_Ai9_R | This gRNA targets the Ai9 and related transgenes, has modified scaffold (Tabebordbar Science 2016) |
Show Experiments (1) |
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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. | ||
Antibody | RRID:AB_2813835 | |||
Antibody | RRID:AB_141607 | |||
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 | AB_141637 | Invitrogen, Donkey anti-rabbit alexa fluor 594 | ||
Antibody | AB_141607 | Invitrogen, Donkey anti-mouse alexafluor 488 | ||
Antibody | RRID:AB_2209751 | |||
Antibody | RRID:AB_10711040 | |||
Vector | AAVcc47-SaCas9-Ai9 | AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter | Asokan Lab | |
Protocol | Gong_Intracranial Injection Procedure for Mice | Procedure for intracranial delivery to mouse brain. | ||
Antibody | AB_2813835 | Abcam, Donkey anti-rat alexa fluour 647 | ||
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_2209751 | Anti-RFP (RABBIT) Antibody | ||
Guide | R2-modified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | sgAi9L | This sgRNA targets the Ai9 and related transgenes | IDT | |
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. | ||
Genome Editor | SauCas9 | |||
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 | ||
Guide | Ai14 gRNA | This sgRNA targets the Ai9 and related transgenes at multiple sites | IDT | |
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_2532994 | Thermo-Fisher, Rat anti-GFAP | ||
Antibody | AB_10711040 | Abcam, mouse anti-NeuN | ||
Antibody | RRID:AB_2532994 | |||
Antibody | RRID:AB_141637 | |||
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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Guide | BCM_ssAAV5-Sp_sgA | This sgRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | BCM_ssAAV5-Sp_sgB | This sgRNA targets the Ai9 and related transgenes | Vector encoded | |
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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Guide | L3-unmodified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | g-loxPbot_C12a | This sgRNA targets the Ai9 and related transgenes at two sites | IDT | |
Guide | sg298 | This sgRNA targets the Ai9 and related transgenes at multiple sites | Synthego | |
Guide | L1-unmodified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | L3-modifed | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | R1-unmodified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | sgAi9L | This sgRNA targets the Ai9 and related transgenes | ||
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 |
Show Experiments (5)
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
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Genome Editor | sNLS-SpCas9-sNLS | SpCas9 with N- and C-terminal SV40 NLS | Aldevron 9212-5MG |
Show Experiments (5)
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
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Guide | g-loxP2_C9 | This sgRNA targets the Ai9 and related transgenes | IDT | |
Guide | L2-modified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | L2-unmodified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | R2-unmodified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | sgAi9R | This sgRNA targets the Ai9 and related transgenes | IDT | |
Guide | BCM_ssAAV5-Sa_sgB | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Guide | R1-modified | This gRNA targets the Ai9 and related transgenes | Vector encoded | |
Vector | AAV9-Ai9-sgRNA1-CB-SaCas9 | AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus | Asokan Lab | |
Vector | AAVcc47-Ai9-sgRNA2-CB-SaCas9 | AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus | Asokan Lab | |
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 |
Show Experiments (3)
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
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Guide | SaLoxP2-unmodified | This gRNA targets the mTmG, Ai9 and related transgenes at two sites | 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 | ||
Vector | AAVcc47-Ai9-sgRNA1-CB-SaCas9 | AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus | Asokan Lab | |
Guide | SaLoxP1-unmodified | This gRNA targets the mTmG, Ai9 and related transgenes at two sites | Vector encoded | |
Guide | SaLoxP2-modified | This gRNA targets the mTmG, Ai9 and related transgenes at two sites | Vector encoded | |
Guide | SaLoxP1-modified | This gRNA targets the mTmG, Ai9 and related transgenes at two sites | Vector encoded | |
Vector | AAV9-Ai9-sgRNA2-CB-SaCas9 | AAV serotype 9 delivering gRNA 2 + 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 | AAVcc47-Ai9-sgRNA1 + sgRNA2 | AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 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 | |
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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Experiment | Testing AAV5 for activation of tdTomato in HEK293T cells | AAV shuttle plasmids expressing SpCas9 and guide RNAs targeting the Ai9 transgene were tested in HEK293T cells by transient transfection. Both delivery and gene editing were detected by fluorescence. | ||
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. | ||
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 | ||
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 | |
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 | |
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 |