Table 1

Subject enrollment characteristics and injection details. Subjects are listed in the order that they were enrolled in the FO study. Eye #1, retina that was initially injected; Eye #2, retina that received the FO injection. All subjects were followed through FOd180. Visual acuity is expressed in LogMAR (log of the minimum angle of resolution). Higher values indicate poorer vision (see Supplementary Methods). Hand motion vision was assigned a conservative LogMAR of 2.6.

Patient IDAge at
readministration
SexFollow-up
after initial
injection (years)
AAV2-hRPE65v2 dose
(vg)/volume (μl)
Visual acuity
(pre/post)
RPE65 mutation
Eye #1Eye #2Eye #1Eye #2
CH1246F2.11.5 × 1011/300
(high/high)
1.5 × 1011/300
(high/high)
2.6/2.162.6/2.0K303X/W431C
CH1127F2.34.8 × 1010/150
(medium/low)
1.5 × 1011/300
(high/high)
0.76/0.770.64/0.58V473D/V473D
NP0129F3.71.5 × 1010/150
(low/low)
1.5 × 1011/300
(high/high)
1.5/1.61.83/1.6E102K/E102K