Table 1.
Capsid engineering details and in vivo tropisms of vectors used in this study
| Capsid name | AAV9 modified sequence | Selection species | Enhanced systemic tropism in the nervous system | Ref. | |||
|---|---|---|---|---|---|---|---|
| VR IV AA452 − 458 | VR VIII AA587 − 590 | Adult Mouse | Adult Marmoset | Infant Macaque | |||
| AAV9 (parent of vectors below) | NGSGQNQ | AQ - - - - - - - AQ | 3 | ||||
| AAV.CAP-B10 | DGAATKN | DGTLAVPFKAQ | Mouse | CNS (neuronal) | CNS (neuronal) | 28 | |
| AAV.CAP-B22 | DGQSSKS | DGTLAVPFKAQ | Mouse | CNS | CNS (neuronal & astrocytic) | 28 | |
| AAV-MaCPNS1 | AQPHEGSSRAQ | Mouse | PNS | CNS & PNS (neuronal & astrocytic) | CNS & PNS (neuronal & astrocytic) | 23 | |
| AAV-MaCPNS2 | AQPNASVNSAQ | Mouse | PNS | CNS & PNS (neuronal & astrocytic) | CNS & PNS (neuronal & astrocytic) | 23 | |
| AAV.CAP-Mac | AQLNTTKPIAQ | Marmoset | CNS (weak endothelial) | CNS (endothelial) | CNS (neuronal) | 21 | |
| AAV9-X1.1 | DGAATKN | AQGNNTRSVAQ | Mouse | CNS (endothelial) | CNS (weak neuronal) | CNS (neuronal) | 20 |
| AAV-BI30 | AQNNSTRGGAQ | Mouse | CNS (endothelial) | 54 | |||