Table 2.
Retinal findings in study 1 after subretinal injection of AAV5-PR2.1-hCNGB3 in phenotypically normal dogs
Group | Animal ID | Eye | Vector dose (vg/eye) | Clinical ophthalmic examination | Histopathologic findings in retina |
---|---|---|---|---|---|
1 | GS135 | OD | 1 × 1010 | Normala | No product-related abnormalities |
OS | 1 × 1010 | Normala | No product-related abnormalities | ||
2 | GS137 | OD | 1 × 1011 | Normala | No product-related abnormalities |
OS | 1 × 1011 | Normala | No product-related abnormalities | ||
3 | GS138 | OD | 1.1 × 1012 | Multifocal retinitis (“black foci,” started at week 5) and focal retinal thinning (hyper-reflective areas by week 14) in bleb region | “Black foci” confirmed as subretinal aggregates of inflammatory cells with loss of photoreceptor inner and outer segments (retinal thinning) |
OS | 1.1 × 1012 | Same as OD | Same as OD | ||
4 | GS139 | OD | 1.1 × 1013 | Multifocal retinitis (“black foci”) and diffuse retinal thinning (hyperreflective areas) in bleb regions started at week 3 | “Black foci” confirmed as subretinal aggregates of inflammatory cells. Retinal thinning due to atrophy of outer nuclear layer. |
OS | 1.1 × 1013 | Same as OD | Same as OD |
Normal except for retinotomy scar, which was seen in all eyes.
OS, left eye; OD, right eye.