Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration

被引:0
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作者
Adeline A. Lau
Kim M. Hemsley
机构
[1] South Australian Health and Medical Research Institute (SAHMRI),Lysosomal Diseases Research Unit, Nutrition and Metabolism Theme
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关键词
Gene therapy; Viral vectors; Lysosomal storage disease; Mucopolysaccharidosis; Central nervous system;
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摘要
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).
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页码:1043 / 1052
页数:9
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