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Article

Adeno‐associated virus gene transfer in Morquio A disease – effect of promoters and sulfatase‐modifying factor 1

28 Jul 2010

Abstract excerpt

Mucopolysaccharidosis (MPS) IVA is an autosomal recessive disorder caused by deficiency of the lysosomal enzyme N-acetylgalatosamine-6-sulfate sulfatase (GALNS), which leads to the accumulation of keratan sulfate and chondroitin 6-sulfate, mainly in bone. To explore the possibility of gene therapy for Morquio A disease, we transduced the GALNS gene into HEK293 cells, human MPS IVA fibroblasts and murine MPS IVA...

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PMID: 20716181
DOI: 10.1111/j.1742-4658.2010.07769.x

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