| Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain |
| Arm1a: BI28 Neuron Specific Promoter, Experimental Animals, Males |
| Qualitative assesment of prevalence of tdTomato-positvie cells throughout the entire brain |
| brain |
| neuron |
| Editing Efficiency |
| 15000001448 |
|
| SaCas9 |
|
|
| L2-modified; L1-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 |
|
| Male |
|
|
| brain |
| 0.25E+11 viral genomes/gram for each virus |
| once |
|
| 5 microliters/gram |
| 4 weeks |
| AAV vector encoded |
|
|
|
| Measured Values |
| Samples Size: | 5 |
| |
| Mean |
5 of 5 present  |
| 1 |
Present  |
| 2 |
Present  |
| 3 |
Present  |
| 4 |
Present  |
| 5 |
Present  |