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Article

Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Apr 2018

Chen Yonghong, Zheng Shujuan, Tecedor Luis, Davidson Beverly L

Abstract excerpt

Sulfamidase (SGSH) deficiency causes mucopolysaccharidosis type IIIA (MPS IIIA), a lysosomal storage disease (LSD) that affects the CNS. In earlier work in LSD mice and dog models, we exploited the utility of adeno-associated viruses (AAVs) to transduce brain ventricular lining cells (ependyma) for secretion of lysosomal hydrolases into the cerebrospinal fluid (CSF), with subsequent distribution of enzyme...

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Identifiers

PMID: 29503202
DOI: 10.1016/j.ymthe.2018.01.010
PMCID: PMC6079371

Genes

SGSH

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