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. Author manuscript; available in PMC: 2022 Oct 6.
Published in final edited form as: Gene Ther. 2022 Apr 6;29(6):368–378. doi: 10.1038/s41434-022-00333-6

Figure 3. Vision loss induced by hND1/m.3460G>A is rescued by gene therapy allele.

Figure 3.

Scatterplot (A) of PERG amplitudes and averaged waveforms (B) of all mice tested in each group showed no significant difference at the baseline between the mice injected with mCherry and hND1/m.3460G>A. however, the difference became statistically significant at 1 month (1m, p=0.011, C, D, two-tailed t-tests ), 3 months (3m, p=0.019, E, F, two-tailed t-tests), 6 months (6m, p=0.020, G, H, two-tailed t-tests), and 12 months (12m, p=0.010, I,J, two-tailed t-tests) after injection. (K). A histogram generated using Generalized Estimating Equation (GEE) analysis showed the mean PERG amplitude significantly decreased with age (p<0.000) but was on average smaller in mutant hND1-injected mice (p<0.001). The time course of age-related PERG changes was different in the two groups (Interaction month × group, p=0.034), indicating the decrease rate was faster in mutant hND1-injected mice than mCherry-injected controls. (L) A box plot using Generalized Estimating Equation (GEE) analysis showed that mice rescued with wildtype hND1(Treated) significantly alleviated the decline in PERG amplitude compared to unrescued control mice (UnTreated) (p=0.028). Month 0 is the baseline, and the following are post injection.