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

21 May 2020

Abstract excerpt

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 promoter. The vectors were delivered intravenously into 4-week-old MPS IVA knockout (KO) and immune tolerant (MTOL)...

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Identifiers

PMID: 32577432
DOI: 10.1016/j.omtm.2020.05.015

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