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Article

Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.

Journal of molecular medicine (Berlin, Germany) - 1 Oct 2017

Lau Adeline A, Hemsley Kim M

Abstract excerpt

The mucopolysaccharidoses (MPS) are a subgroup of lysosomal storage disorders that are caused by mutations in the genes involved in glycosaminoglycan breakdown. Multiple organs and tissues are affected, including the central nervous system. At present, hematopoietic stem cell transplantation and enzyme replacement therapies are approved for some of the (non-neurological) MPS. Treatments that effectively...

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PMID: 28660346
DOI: 10.1007/s00109-017-1562-0

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