Article
Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.
Lancet (London, England) - 7 Sept 2024
Yang Paul, Pardon Laura P, Ho Allen C, Lauer Andreas K, Yoon Dan, Boye Shannon E, Boye Sanford L, Roman Alejandro J, Wu Vivian, Garafalo Alexandra V, Sumaroka Alexander, Swider Malgorzata, Viarbitskaya Iryna, Aleman Tomas S, Pennesi Mark E, Kay Christine N, Fujita Kenji P, Cideciyan Artur V
Abstract excerpt
BACKGROUND: Leber congenital amaurosis 1 (LCA1), caused by mutations in GUCY2D, is a rare inherited retinal disease that typically causes blindness in early childhood. The aim of this study was to evaluate the safety and preliminary efficacy of ascending doses of ATSN-101, a subretinal AAV5 gene therapy for LCA1. METHODS: 15 patients with genetically confirmed biallelic mutations in GUCY2D were included in this...
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