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Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity

Pompe disease is a severe disorder caused by loss of acid α‐glucosidase (GAA), leading to glycogen accumulation in tissues and neuromuscular and cardiac dysfunction. Enzyme replacement therapy is the only available treatment. AT845 is an adeno‐associated viral vector designed to express human GAA sp...

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Detalles Bibliográficos
Autores principales:	Eggers, Michelle, Vannoy, Charles H, Huang, Jianyong, Purushothaman, Pravinkumar, Brassard, Jacqueline, Fonck, Carlos, Meng, Hui, Prom, Mariah J, Lawlor, Michael W, Cunningham, Justine, Sadhu, Chanchal, Mavilio, Fulvio
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8749482/ https://www.ncbi.nlm.nih.gov/pubmed/34850579 http://dx.doi.org/10.15252/emmm.202113968

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