Table 1.
Retinal thicknesses with various promoters
| Promoter | Genotype | Mean NFL to RPE thickness (μm) |
Mean OPL to RPE thickness (μm) |
||
|---|---|---|---|---|---|
| 2 months | 4 months | 2 months | 4 months | ||
| CASI | WT | 222.46 | 210.75 | 125.42 | 119.04 |
| Nnmnat1V9M/V9M | 215.95 | 191.6 | 119.1 | 106.45 | |
| hGRK1 | WT | 222.53 | 217.27 | 125.69 | 122.97 |
| Nnmnat1V9M/V9M | 201.03 | 182.89 | 102.93 | 93.5 | |
| ProC1 | WT | 222.92 | 217.56 | 128.06 | 124.72 |
| Nnmnat1V9M/V9M | 203.88 | 182.56 | 109.03 | 95.34 | |
| VMD2 | WT | 214.25 | 210.67 | 124.08 | 123.25 |
| Nnmnat1V9M/V9M | 163.25 | 146.34 | 71.7 | 58.9 | |
| ProC3 | WT | 221.79 | 219.9 | 124.29 | 124.81 |
| Nnmnat1V9M/V9M | 194.25 | 180.6 | 101.5 | 93.34 | |
| ProD5 | WT | 222.36 | 217.36 | 126.32 | 124.07 |
| Nnmnat1V9M/V9M | 201.2 | 185.41 | 108.83 | 101.88 | |
| Untreated | WT | 226.61 | 221.99 | 130.47 | 128.85 |
| Nnmnat1V9M/V9M | 154.8 | 125.25 | 61.66 | 39.09 | |
Mean retinal thickness measured from the nerve fiber layer (NFL) to the retinal pigment epithelium (RPE) or the outer plexiform layer (OPL) to the RPE at 2 months and 4 months of age. Measurements represent the thickness in the inferior region of the retina (closest to the injection site). All treated eyes were treated with scAAV9 with the respective promoter driving expression of hNMNAT1. The virus was delivered at 1E9 vg/μL and co-injected with AAV9 CMV.GFP at a titer of 1E8 vg/μL. WT, wild type.