Please use this identifier to cite or link to this item: http://hdl.handle.net/2440/111828
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Type: Journal article
Title: Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration
Author: Lau, A.
Hemsley, K.
Citation: Journal of Molecular Medicine, 2017; 95(10):1043-1052
Publisher: Springer Verlag
Issue Date: 2017
ISSN: 0946-2716
1432-1440
Statement of
Responsibility: 
Adeline A. Lau, Kim M. Hemsley
Abstract: 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 ameliorate the neurological aspects of the disease are being assessed in clinical trials. This review will focus on the recent outcomes and planned viral vector-mediated gene therapy clinical trials, and the pre-clinical data that supported these studies, for MPS-I (Hurler/Scheie syndrome), MPS-II (Hunter syndrome), and MPS-IIIA and -IIIB (Sanfilippo syndrome).
Keywords: Central nervous system; Gene therapy; Lysosomal storage disease; Mucopolysaccharidosis; Viral vectors
Rights: © Springer-Verlag GmbH Germany 2017
RMID: 0030084930
DOI: 10.1007/s00109-017-1562-0
Appears in Collections:Medicine publications

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