Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse

Niemann-Pick disease (NPD) is caused by the loss of acid sphingomyelinase (ASM) activity, which results in widespread accumulation of undegraded lipids in cells of the viscera and CNS. In this study, we tested the effect of combination brain and systemic injections of recombinant adeno-associated vi...

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Detalhes bibliográficos
Main Authors: Passini, Marco A., Bu, Jie, Fidler, Jonathan A., Ziegler, Robin J., Foley, Joseph W., Dodge, James C., Yang, Wendy W., Clarke, Jennifer, Taksir, Tatyana V., Griffiths, Denise A., Zhao, Michael A., O'Riordan, Catherine R., Schuchman, Edward H., Shihabuddin, Lamya S., Cheng, Seng H.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2007
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1874225/
https://ncbi.nlm.nih.gov/pubmed/17517638
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0703509104
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