Title : Evaluation of HIV-1 derived lentiviral vectors as transductors of Mucopolysaccharidosis type IV a fibroblasts.

Pub. Date : 2021 May 15

PMID : 33636292






1 Functional Relationships(s)
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1 Mucopolysaccharidosis type IVA (MPS IVA) is a lysosomal storage disease produced by the deficiency of the N-acetylgalactosamine-6-sulfate sulfatase (GALNS) enzyme, leading to glycosaminoglycans (GAGs) accumulation. Glycosaminoglycans galactosamine (N-acetyl)-6-sulfatase Homo sapiens