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PICO
Question: Among children treated with gene therapy for Leber’s congenital amaurosis, are initial visual improvements sustained?
Question type: Treatment
Study design: Case series
Investigators from multiple institutions report on the long-term follow-up of patients treated in a gene therapy trial for Leber’s congenital amaurosis (LCA). LCA represents a group of typically autosomal recessive disorders wherein progressive photoreceptor loss results in childhood-onset blindness. LCA caused by mutations in the gene coding for retinal pigment epithelium (RPE) protein 65 kDA (RPE65) was the initial target for gene therapy. For the trial, a single adeno-associated …
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