Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A
Maria Grazia Biferi, Mathilde Cohen-Tannoudji, Andrea García-Silva, Olga Souto-Rodríguez et autres
Fabry disease is a rare X-linked disorder affecting α-galactosidase A, a rate-limiting enzyme in lysosomal catabolism of glycosphingolipids. Current treatments present important limitations, such as low half-life and limited distribution, which gene therapy can overcome. The aim of this work was to …
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27 citations
Molecular Therapy — Methods & Clinical Development