Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice

Mucopolysaccharidosis (MPS) IIIA is a neuropathic lysosomal storage disease caused by deficiency in N-sulfoglucosamine sulfohydrolase (SGSH). Genome-wide gene expression microarrays in MPS IIIA mice detected broad molecular abnormalities (greater than or equal to twofold, false discovery rate ≤10) i...

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Bibliographic Details
Published in:Mol Ther
Main Authors: Duncan, F Jason, Naughton, Bartholomew J, Zaraspe, Kimberly, Murrey, Darren A, Meadows, Aaron S, Clark, Kelly Reed, Newsom, David E, White, Peter, Fu, Haiyan, McCarty, Douglas M
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2015
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4395798/
https://ncbi.nlm.nih.gov/pubmed/25592334
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.9
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