Project: BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice
PI:
Jason D Heaney, PhD
Summary of data submissions:
- Data for 1 validation experiments were submitted on 2021-03-30 SCGE ID:1040
- Data for 1 validation experiments were submitted on 2021-08-08 SCGE ID:1041
- Data for 1 validation experiments were submitted on 2021-03-30 SCGE ID:1042
- Data for 1 validation experiments were submitted on 2021-09-07 SCGE ID:1043
- Data for 1 validation experiments were submitted on 2022-03-28 SCGE ID:1091
Submissions Details
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VALIDATION - BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Asokan Validation (Asokan, Aravind)
SCGE ID:1040 - Submission
Date: 2021-03-30
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| Experiment Name | Type | Description |
|---|---|---|
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Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Original Experiment/s that are being validated:
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In Vivo | Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues. |
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VALIDATION - BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin)
SCGE ID:1041 - Submission
Date: 2021-08-08
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| Experiment Name | Type | Description |
|---|---|---|
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Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Original Experiment/s that are being validated:
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In Vivo | 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 |
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VALIDATION - BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Gao Validation (Gao, Guang-Ping )
SCGE ID:1042 - Submission
Date: 2021-03-30
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| Experiment Name | Type | Description |
|---|---|---|
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Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Original Experiment/s that are being validated:
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In Vivo | 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 |
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VALIDATION - BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - McCray Validation (McCray Jr., Paul)
SCGE ID:1043 - Submission
Date: 2021-09-07
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| Experiment Name | Type | Description |
|---|---|---|
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Independent validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
Original Experiment/s that are being validated:
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In Vivo | 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 |
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VALIDATION - BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Chaikof Validation (Chaikof, Elliot L. )
SCGE ID:1091 - Submission
Date: 2022-03-28
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| Experiment Name | Type | Description |
|---|---|---|
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Independent validation of Chaikof delivery platform using virus-like particle (VLP) delivery to the mouse liver
Original Experiment/s that are being validated:
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In Vivo | Virus-like particles carrying a CRISPR/Cas base editor and a guide RNA targeting the PSCK9 locus were injected i.v. into male and female mice. One week after injection, the mice were dissected, and genomic DNA isolated from a panel of organs. Targeted NGS was performed to evaluate the degree of editing at the PCSK9 locus in the liver (primary target), and non-target organs. Two potential off-target editing sites (OT6 and OT7) were also sequenced. |