Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration

Title
Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration
Authors
Keywords
Gene therapy, Viral vectors, Lysosomal storage disease, Mucopolysaccharidosis, Central nervous system
Journal
JOURNAL OF MOLECULAR MEDICINE-JMM
Volume 95, Issue 10, Pages 1043-1052
Publisher
Springer Nature
Online
2017-06-29
DOI
10.1007/s00109-017-1562-0

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