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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Bibliographic Details
Published in:Mol Ther Methods Clin Dev
Main Authors: Sawamoto, Kazuki, Karumuthil-Melethil, Subha, Khan, Shaukat, Stapleton, Molly, Bruder, Joseph T., Danos, Olivier, Tomatsu, Shunji
Format: Artigo
Language:Inglês
Published: American Society of Gene & Cell Therapy 2020
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7301175/
https://ncbi.nlm.nih.gov/pubmed/32577432
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.05.015
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