Figure 6 | Gene Therapy

Figure 6

From: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

Figure 6

Structural and functional assessments of WT mouse and rabbit retinas following subretinal injection of AAV2/5-OPTIRPE65. ERG responses and retinal thickness measurements of eyes injected with AAV2/5-OPTIRPE65 (n=10 for mice, n=9 for rabbits) and untreated control eyes (n=4 for mice, n=9 for rabbits) of young adult WT mice. Mean±s.d. scotopic (at 0.01 Cds per m2) and photopic (at 10 Cds per m2) ERG b-wave amplitudes of mice retinas at 9 months (a) or rabbit retinas at 8 weeks (d) are shown. The Y axis denotes μV values of electrical conductivity. Dose: 4 × 109 vg per eye in mice, 2 × 1011 vg per eye in rabbits. Mean±s.d. photoreceptor cell layer thickness measurements from treated and untreated eyes are shown (b for mice, e for rabbits). Representative images of mouse (c) and rabbit (f) retinal morphology: GCL, ganglion cell layer; INL, inner nuclear layer; outer nuclear layer; IS/OS, inner segments/outer segments; RPE.

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