Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain |
Arm 2a: BI28 Astrocyte Specific Promoter, Experimental Animals, Females |
Identification of of GFP-positive nuclei (green) |
brain |
astrocyte |
Delivery Efficiency |
15000001459 |
|
SaCas9 |
|
|
L1-modified; L2-modified |
BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2; BI28:AAV-GFAP-SaCas9-WPRE3-pA; |
|
Mouse |
B6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J |
|
|
brain |
0.25E+11 viral genomes/gram for each virus |
once |
|
5 microliters/gram |
4 weeks |
ssDNA, packaged in two AAVs with astrocyte-specific promoter |
|
|
|
Measured Values |
Samples Size: | 6 |
|
Mean |
6 of 6 present  |
1 |
present  |
2 |
present  |
3 |
present  |
4 |
present  |
5 |
present  |
6 |
present  |