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

Internet

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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