The present application provides compositions for treatment of Friedreich's Ataxia (FA). These include, but are not limited to, nucleic acid constructs and recombinant AAV7 vectors comprising a human frataxin 5' untranslated region (5'UTR FXN) and a human frataxin (FXN).
Saturday, May 11, 2024
1. WO2024097772 - COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA
N.º de publicación
WO/2024/097772. Fecha de publicación
10.05.2024. Nº de la solicitud internacional
PCT/US2023/078373. Fecha de presentación internacional
01.11.2023.
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