Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice |
ssAAV5-spCas9 + ssAAV5-sgA+sgB-U1A.GFP, Ai9 - SauSpyCas9, female |
Fluorescent imaging for tdTomato+ cells |
duodenum |
Editing Efficiency |
15000001072 |
|
SpCas9 |
|
|
BCM_ssAAV5-Sp_sgA; BCM_ssAAV5-Sp_sgB |
ssAAV5-sgA+sgB-U1A.GFP; ssAAV5-spCas9; |
|
Mouse |
B6.Cg-Gt(ROSA)26Sor^em1(CAG-tdTomato)JaHe |
|
Intratracheal Intubation |
lung airway epithelium |
1.7E11 viral genomes |
once |
|
40 microliters |
4 weeks |
virus in sterile saline |
|
|
|
Measured Values |
Samples Size: | 5 |
|
Mean |
5 no red cells  |
1 |
no red cells  |
2 |
no red cells  |
3 |
no red cells  |
4 |
no red cells  |
5 |
no red cells  |