Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A-Reference-Cited by-同舟云学术

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

Published:2021-03 Issue: Volume:20 Page:1-17
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

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

Funder

Instituto de Salud Carlos III

European Commission

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference51 articles.

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4. Relationship between X-inactivation and clinical involvement in Fabry heterozygotes. Eleven novel mutations in the α-galactosidase A gene in the Czech and Slovak population;Dobrovolny;J. Mol. Med. (Berl.),2005

5. Long Term Treatment with Enzyme Replacement Therapy in Patients with Fabry Disease;Oder;Nephron,2016

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