A study conducted in the United Kingdom showed that young children with inherited retinal diseases who underwent gene therapy using voretigene neparvovec experienced greater improvements in vision compared to adults. The treatment targets a specific gene mutation in retinitis pigmentosa and Leber congenital amaurosis. Results indicated an average improvement in visual acuity and functional tests in children who received the treatment early. The study population included adults who also showed some benefit, although caution is advised due to irreversible damage in adults. The debate on whether to detach the fovea during the gene therapy procedure is ongoing, with varying approaches based on individual cases.
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In Gene Therapy for RP, LCA, the Earlier, the Better
