Table 1.
Injection Material Details | Dose (Total Genome Copies Per Eye) | Abbreviated Name |
---|---|---|
AAV diluent: PBS with 0.001% PF68 | Not applicable | Sham |
GRK1.GFP.pA: GRK1 promoter driving GFP expression | 2E+10 | GFP |
GRK1.ABC: GRK1 promoter with the 5′ portion of ABCA4 coding sequence | 2E+10 | 5′ vector high dose |
2E+09 | 5′ vector low dose | |
CA4.WPRE.pA: 3′ portion of ABCA4 coding sequence with WPRE and polyA signal | 1E+10 | 3′ vector |
GRK1.ABC + CA4.WPRE.pA: 1:1 mix of 5′ and 3′ vectors | 2E+10 | Dual vector high dose |
2E+09 | Dual vector low dose |
PF68, pluronic PF-68.