Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A

Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A

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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Bibliographic Details
Main Authors: Maria Grazia Biferi, Mathilde Cohen-Tannoudji, Andrea García-Silva, Olga Souto-Rodríguez, Irene Viéitez-González, Beatriz San-Millán-Tejado, Andrea Fernández-Carrera, Tania Pérez-Márquez, Susana Teijeira-Bautista, Soraya Barrera, Vanesa Domínguez, Thibaut Marais, África González-Fernández, Martine Barkats, Saida Ortolano
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Fabry disease
lysosomal storage disorders
adeno asociated virus-9
gene therapy
blood brain barrier
gene transfer
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120302205

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http://www.sciencedirect.com/science/article/pii/S2329050120302205

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