Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease

Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease

We developed a novel adeno-associated virus 5 gene therapy (AAV5-GLA) expressing human alpha-galactosidase A (GLA) under the control of a novel, small and strong, liver-restricted promoter. We assessed the preclinical potential of AAV5-GLA for treating Fabry disease, an X-linked hereditary metabolic...

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Main Authors: Jolanda M.P. Liefhebber, Giso Brasser, Elisabeth A. Spronck, Roelof Ottenhoff, Lieke Paerels, Maria J. Ferraz, Lukas K. Schwarz, Nikoleta Efthymiopoulou, Chi-Lin Kuo, Paula S. Montenegro-Miranda, Melvin M. Evers, Johannes M.F.G. Aerts, Ying Poi Liu
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
glycosphingolipids
lysosomal storage diseases
Macaca fascicularis
metabolism
mice
knockout
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050124001918
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