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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Biferi, Maria Grazia, Cohen-Tannoudji, Mathilde, García-Silva, Andrea, Souto-Rodríguez, Olga, Viéitez-González, Irene, San-Millán-Tejado, Beatriz, Fernández-Carrera, Andrea, Pérez-Márquez, Tania, Teijeira-Bautista, Susana, Barrera, Soraya, Domínguez, Vanesa, Marais, Thibaut, González-Fernández, África, Barkats, Martine, Ortolano, Saida
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7725667/
https://ncbi.nlm.nih.gov/pubmed/33335943
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.10.016
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