Table 1.

Clinical trial of gene therapy for GUCY2D-LCA

	Age at baseline (y)/Sex	GUCY2D mutations Allele 1/Allele 2	Treated Eye	Injection sitea	Concentration (vg/mL)	Total volumeb (μL)	Entry VA, (logMAR)c		Entry FST (log10)d
							Study eye	Control eye	Study eye	Control eye
P1	45/F	c.2943delG, p.Ser981del1bpe,f/ c.2302C>T, p.Arg768Trpe,f,g	Right	Macula	3.3 × 1010	300	2.6	2.6	4.3	4.5
P2	35/M	c.1780_1783delCTCT, p.Leu594Thrfs∗42f/ c.3160T>A, p.Phe1054Ilef	Right	Superior retina	3.3 × 1010	200	1.25	1.19	4.4	4.2
P3	23/F	c.1978C>T, p.Arg660∗h/ c.2984G>A, p.Arg995Glni	Left	Macula	3.3 × 1010	300	1.27	0.65	5.2	5.3

^aPlanned for the macula; the bleb in P2 was unable to be raised in the macula and was in the superior retina instead.

^bIntended subretinal volume was 300 μL, but P2 received approximately 200 μL, the estimate at time of surgery.

^cBest-corrected visual acuity at baseline visit, measured from back-lit Early Treatment Diabetic Retinopathy Study charts adjusted for distance (0.00 logMAR = 20/20 Snellen VA at 4 m) in P2 and P3; BVRT (Berkeley Rudimentary Vision Test) was used for P1.

^dRod sensitivity (in log10 1/(phot-cd.m-2)) at baseline visit, measured by full-field stimulus testing (FST) with blue stimulus.

^eJacobson et al. (2013).

^fJacobson et al. (2017b).

^gHanein et al. (2004).

^hLi et al. (2009).

ⁱNovel.