Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy

Published 2015 View Full Article

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Title
Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy
Authors
Keywords
Sandhoff disease, Lysosomal storage disease, Gene therapy, Bone, Heart, GM2 gangliosidosis
Journal
MOLECULAR GENETICS AND METABOLISM
Volume 116, Issue 1-2, Pages 80-87
Publisher
Elsevier BV
Online
2015-05-08
DOI
10.1016/j.ymgme.2015.05.003

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