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Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model

Mucopolysaccharidosis type IVA (MPS IVA) is due to the deficiency of GALNS (N-acetylgalactosamine 6-sulfate sulfatase) and is characterized by systemic skeletal dysplasia. We have evaluated adeno-associated virus 8 (AAV8) vectors expressing different forms of human GALNS under a liver-specific promo...

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Detalles Bibliográficos
Autores principales:	Sawamoto, Kazuki, Karumuthil-Melethil, Subha, Khan, Shaukat, Stapleton, Molly, Bruder, Joseph T., Danos, Olivier, Tomatsu, Shunji
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7301175/ https://www.ncbi.nlm.nih.gov/pubmed/32577432 http://dx.doi.org/10.1016/j.omtm.2020.05.015

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