Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain |
Arm 2a: BI28 Astrocyte Specific Promoter, Experimental Animals, Males |
Quantitative assessment of tdTomato-positive cells per DAPI-stained nucleus in a specific brain region |
dorsal plus ventral thalamus |
astrocyte |
Editing Efficiency |
15000001455 |
|
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 |
AAV vector encoded |
|
|
|
Measured Values |
Samples Size: | 6 |
|
Mean |
43.4  |
1 |
53.5  |
2 |
29.5  |
3 |
29.2  |
4 |
53.8  |
5 |
46.4  |
6 |
48.0  |