Experiment: Large animal testing of AAV9 vs AAVcc47 (Asokan lab's novel capsid) via intravenous administration to rhesus macaques
PI: Aravind Asokan, PhD
Description: Phase I, AAV9 (n=4) and AAVcc47 (n=4) were administered via intravenous injection in rhesus macaques and organs were harvested 4 weeks post injection.
Delivery Assays:
Parent Project: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing - submission 3
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Other experiments in this project: 8
- Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
- Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
- Cre Recombinase dose escalation study in Ai9 mice
- Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (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 efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
- Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
- Large animal testing of AAV9 vs AAVcc47 (Asokan lab's novel capsid) via intramuscular administration to rhesus macaques
Download: Submitted files
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Results |
| Delivery Efficiency | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Condition | Qualifier | Tissue | Cell Type | Age | Editor | Delivery | Target Locus | Guide | Vector | Dosage | Injection Frequency | SaCas9/globin copies | Image |
| AAV9 | liver | ~3-4 months of age | SaCas9 | AAV | DMD | JCR183 | AAV9-SaCas9/guide DMD | 1. 3e13vg/kg (1~1.5kg body weight) | Once | ||||
| AAV9 | left | quadriceps femoris | ~3-4 months of age | SaCas9 | AAV | DMD | JCR183 | AAV9-SaCas9/guide DMD | 1. 3e13vg/kg (1~1.5kg body weight) | Once | |||
| AAV9 | heart | ~3-4 months of age | SaCas9 | AAV | DMD | JCR183 | AAV9-SaCas9/guide DMD | 1. 3e13vg/kg (1~1.5kg body weight) | Once | ||||
| AAVcc47 | liver | ~3-4 months of age | SaCas9 | AAV | DMD | JCR183 | AAVcc47-SaCas9/guide DMD | 1. 3e13vg/kg (1~1.5kg body weight) | Once | ||||
| AAVcc47 | left | quadriceps femoris | ~3-4 months of age | SaCas9 | AAV | DMD | JCR183 | AAVcc47-SaCas9/guide DMD | 1. 3e13vg/kg (1~1.5kg body weight) | Once | |||
| AAVcc47 | heart | ~3-4 months of age | SaCas9 | AAV | DMD | JCR183 | AAVcc47-SaCas9/guide DMD | 1. 3e13vg/kg (1~1.5kg body weight) | Once | ||||
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