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Article

Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations

14 Apr 2021

Abstract excerpt

A first-in-human clinical trial of gene therapy in Leber congenital amaurosis due to mutations in the GUCY2D gene is underway, and early results are summarized. A recombinant adeno-associated virus serotype 5 (rAAV5) vector carrying the human GUCY2D gene was delivered by subretinal injection to one eye in three adult patients with severe visual loss, nystagmus, but preserved retinal structure. Safety and efficacy...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 33997691
DOI: 10.1016/j.isci.2021.102409

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