Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates

No treatment is currently available for mucopolysaccharidosis (MPS) IIIB, a neuropathic lysosomal storage disease caused by autosomal recessive defect in α-N-acetylglucosaminidase (NAGLU). In anticipation of a clinical gene therapy treatment for MPS IIIB in humans, we tested the rAAV9-CMV-hNAGLU vec...

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Bibliografiske detaljer
Main Authors: Murrey, Darren A., Naughton, Bartholomew J., Duncan, F. Jason, Meadows, Aaron S., Ware, Tierra A., Campbell, Katie J., Bremer, William G., Walker, Christopher M., Goodchild, Laurie, Bolon, Brad, La Perle, Krista, Flanigan, Kevin M., McBride, Kim L., McCarty, Douglas M., Fu, Haiyan
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2014
Fag:
Pre-Clinical Safety Study
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4124586/
https://ncbi.nlm.nih.gov/pubmed/24720466
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2013.208
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