Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1

Lysosomal storage diseases (LSDs) are debilitating neurometabolic disorders for most of which long-term effective therapies have not been developed. Gene therapy is a potential treatment but a critical barrier to treating the brain is the need for global correction. We tested the efficacy of cistern...

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Bibliographic Details
Published in:Mol Ther
Main Authors: Yoon, Sea Young, Bagel, Jessica H, O'Donnell, Patricia A, Vite, Charles H, Wolfe, John H
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2016
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4754545/
https://ncbi.nlm.nih.gov/pubmed/26354342
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.168
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