A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB

No treatment is currently available for mucopolysaccharidosis (MPS) IIIB, a neuropathic lysosomal storage disease due to defect in α-N-acetylglucosaminidase (NAGLU). In preparation for a clinical trial, we performed an IND-enabling GLP-toxicology study to assess systemic rAAV9-CMV-hNAGLU gene delive...

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Bibliografski detalji
Izdano u:Hum Gene Ther Clin Dev
Glavni autori: Meadows, Aaron S., Duncan, F. Jason, Camboni, Marybeth, Waligura, Kathryn, Montgomery, Chrystal, Zaraspe, Kimberly, Naughton, Bartholomew J., Bremer, William G., Shilling, Christopher, Walker, Christopher M., Bolon, Brad, Flanigan, Kevin M., McBride, Kim L., McCarty, Douglas M., Fu, Haiyan
Format: Artigo
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc. 2015
Teme:
Research Articles
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4733322/
https://ncbi.nlm.nih.gov/pubmed/26684447
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2015.132
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