Table 3.
Group | Animal ID | Eye | Vector dose (vg/eye) | Cone ERG (μV) | Clinical ophthalmic examination | Histopathologic findings in retina |
---|---|---|---|---|---|---|
1 | GS225 | OD | 7.5 × 109 | 0 | Focal retina thinning | Focal inner and outer nuclear layer attenuation |
OS | 7.5 × 109 | 0 | Normala | Focal disruption of retinal architecture; minimal neuroretinal accumulation of mononuclear inflammatory cells | ||
2 | GS226 | OD | 7.5 × 1010 | 2.12 | Retinal fold; focal retinal thinning | Focal detachment with associated focal neuroretinal (predominantly perivascular) accumulation of mononuclear inflammatory cells; rare vitreal inflammatory cells |
OS | 7.5 × 1010 | 2.90 | Retinal fold; focal retinal thinning | Moderate neuroretinal (predominantly perivascular) accumulation of mononuclear inflammatory cells; rare vitreal inflammatory cells | ||
GS229 | OD | 7.5 × 1010 | 13.00 | Normala | Multifocal, predominantly perivascular mononuclear inflammation of the neuroretina | |
OS | 7.5 × 1010 | 19.10 | Discoloration at injection site | Focal neuroretinal atrophy (retinotomy scar) | ||
3 | GS228 | OD | 7.5 × 1011 | 0 | Multifocal chorioretinitis started at week 7 | No significant lesions |
OS | 7.5 × 1011 | 0 | Multifocal chorioretinitis started at week 4 | Locally extensive retinal attenuation with neuroretinal parenchymal and perivascular accumulation of mononuclear inflammatory cells; rare vitreal inflammatory cells | ||
GS233 | OD | 7.5 × 1011 | 3.41 | Multifocal chorioretinitis started at week 6 | Focal retinal detachment with neuroretinal parenchymal and perivascular mononuclear inflammation; retinal pigment epithelium loss and associated inflammation | |
OS | 7.5 × 1011 | 2.73 | Multifocal chorioretinitis started at week 6 | Locally extensive marked retinal atrophy; perivascular mononuclear inflammation |
Each animal received a 0.15 mL subretinal injection of AAV5-PR2.1-hCNGB3 in both eyes (OD and OS).
Normal except for retinotomy scar which was seen in all eyes.
ERG, electroretinogram.