Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. |
cc47-SaCas9, Female |
Fluorescent imaging for tdTomato+ cells |
extensor digitorum longus |
Editing Efficiency |
15000001276 |
|
SauCas9 |
|
|
Ai9 SaCas9 Guide A; Ai9 SaCas9 Guide B |
AAVcc47-SaCas9-Ai9; |
|
Mouse |
B6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J |
|
intravenous, tail vein |
liver and heart |
1.0x10^14 vg copies/kg (vg/kg) (2.0x10^12/20g mouse) |
once |
|
200 microliters |
4 weeks |
AAV vector encoded |
|
|
|
Measured Values |
Samples Size: | 5 |
|
Mean |
1 One red cell; 4 No red cells  |
1 |
One red cell  |
2 |
No red cells  |
3 |
No red cells  |
4 |
No red cells  |
5 |
No red cells  |