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|Title:||Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration|
|Citation:||Journal of Molecular Medicine, 2017; 95(10):1043-1052|
|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|
|Appears in Collections:||Medicine publications|
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