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Spatial and temporal resolution of the photoreceptors rescue dynamics after treatment with voretigene neparvovec

British Journal of Ophthalmology Jan 24, 2021

Stingl K, Kempf M, Bartz-Schmidt KU, et al. - Since voretigene neparvovec is a gene therapeutic agent for the treatment of retinal dystrophies caused by bi-allelic RPE65 mutations, researchers sought to examine the longitudinal retinotopic change in the rod and cone photoreceptor function with novel methods. Seven eyes of five patients (14, 21, 23, 24, 36 years, 1 male, 4 females) with bi-allelic RPE65 mutations were treated with voretigene neparvovec. The protocols presented allow the individual spatial and temporal effects of gene therapy effects to be evaluated. In addition, parameters that correlated with the success of the therapy were explored. Chromatic pupil campimetry and dark-adapted chromatic perimeter present new and fast ways of assessing functional changes in rod and cone function retinotopic maps, measuring complementary aspects of retinal function.

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