Genetically modified cones rescue visual function in children

Gene therapy can restore retinal signals absent since birth in animal models, yet the translation to humans remains challenging. An unresolved possibility is that meaningful retinal rescue already occurs in humans, but its impact is masked by downstream neural bottlenecks shaped by long-term deprivation. Tracing restored signals through the visual system is therefore essential to distinguish rescue and constraints. We address this challenge in CNGA3-associated achromatopsia, in which retinal cone photoreceptors are present but non-functional from birth. We used tests specifically targeting con

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