Table 1.
Summary of the effect of the different vectors after subretinal injection in Fam161atm1b/tm1b mice.
| Vector | Expression | Rescue | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Regulatory elements | cDNA | Dose (GC/eye) | IHC labeling | Figure | U-exM | Figure | ONL thickness | Figure | ERG | Figure |
| IRBP-GRK1 | GFP | 1.3 x 109 | Entire PR/+++ | 1E | Nd | Nd | Nd | |||
| IRBP-GRK1 | HS | 1 x 1010 | Entire PR/+++ | 2G | Nd | Improved | 2J | Not improved | 2A | |
| IRBP-GRK1 | HL | 1 x 1010 | Entire PR/+++ | 2H | Nd | Improved | 2L | Not improved | 2A | |
| IRBP-GRK1 | HS + HL | 1 x 1010 | Entire PR/+++ | 2I | Longer CC | 3, 5F, App. Fig. 4 | Improved | 2N | Not improved | 2B–D |
| IRBP-GRK1 | HS + HL | 1 x 109 | Entire PR/++ | App. Fig. 2 | Nd | Nd | Nd | |||
| IRBP-GRK1 | HS + HL | 1 x 108 | Entire PR/+ | App. Fig. 2 | Nd | Nd | Nd | |||
| FCBR2-F0.4 | GFP | 1 x 1010 | PR+RPE/++ | 1G | Nd | Nd | Nd | |||
| FCBR2-F0.4 | HS | 1 x 1010 | Entire PR/++ | 4A | Nd | Nd | Nd | |||
| FCBR1-F0.4 | GFP | 1 x 1010 | PR/+ | 1F | Nd | Not improved | 4H, 6 | Not improved | 7A–C | |
| FCBR1-F0.4 | HS | 1 x 1010 | CC + entire PR/+ | 4B | Nd | Nd | Nd | |||
| FCBR1-F0.4 | HS | 1 x 1011 | CC + entire PR/+ | 4D, App. Fig. 3B | CC + entire PR | 5B,F, App. Fig. 4 | Improved | 4H, 6 | Not improved | 7A |
| FCBR1-F0.4 | HL | 1 x 1010 | CC/+ | 4C | Nd | Nd | Nd | |||
| FCBR1-F0.4 | HL | 1 x 1011 | CC/++ | 4E, App. Fig. 3A | CC | 5C,F, Aupp. Fig. 4 | Improved | 4H, 6 | Weakly improved | 7B |
| FCBR1-F0.4 | HS + HL | 1 x 1011 | CC/+++ | 4F,G, App. Fig. 3C | CC | 5D,F, App. Fig. 4 | Improved | 4H, 6 | Improved | 7C–G |
+ weak expression, ++ medium expression, +++ high level of expression, CC connecting cilium, PR photoreceptor, nd not determined.