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

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
Main Authors: Kazuki Sawamoto, Subha Karumuthil-Melethil, Shaukat Khan, Molly Stapleton, Joseph T. Bruder, Olivier Danos, Shunji Tomatsu
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
MPS IVA
GALNS
AAV
liver targeting
keratan sulfate
skeletal dysplasia
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301029

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http://www.sciencedirect.com/science/article/pii/S2329050120301029

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