Title : Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modifying factor 1.

Pub. Date : 2010 Sep

PMID : 20716181






1 Functional Relationships(s)
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1 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. Chondroitin Sulfates galactosamine (N-acetyl)-6-sulfatase Homo sapiens