| Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain |
| Arm 2b: BI28 Astrocyte Specific Promoter, Positive Delivery Control Animals, Males |
| Identification of of GFP-positive nuclei (green) |
| brain |
| astrocyte |
| Delivery Efficiency |
| 15000001460 |
|
| SaCas9 |
|
|
|
| BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2; |
|
| Mouse |
| B6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J |
|
| Male |
|
|
| brain |
| 0.25E+11 viral genomes/gram for each virus |
| once |
|
| 5 microliters/gram |
| 4 weeks |
| AAV vector encoded |
|
|
|
| Measured Values |
| Samples Size: | 3 |
| |
| Mean |
3 of 3 present  |
| 1 |
present  |
| 2 |
Present  |
| 3 |
Present  |