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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Detalhes bibliográficos
Publicado no:Hum Gene Ther Clin Dev
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2015
Assuntos:
Research Articles
Acesso em linha: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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