Safety Study of Adeno-Associated Virus Serotype 2-Mediated Human Acid Sphingomyelinase Expression in the Nonhuman Primate Brain

Niemann-Pick disease is a lysosomal storage disorder resulting from inherited deficiency in acid sphingomyelinase (ASM). Use of adeno-associated virus serotype 2 (AAV2) to deliver human acid sphingomyelinase (hASM) is currently being explored as a means to treat the devastating neurological features...

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Main Authors: Salegio, Ernesto A., Samaranch, Lluis, Jenkins, Russell W., Clarke, Christopher J., Lamarre, Clementine, Beyer, Janine, Kells, Adrian P., Bringas, John, Sebastian, Waldy San, Richardson, R. Mark, Rosenbluth, Kate H., Hannun, Yusuf A., Bankiewicz, Krystof S., Forsayeth, John
格式: Artigo
語言:Inglês
出版: Mary Ann Liebert, Inc. 2012
主題:
Research Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3413900/
https://ncbi.nlm.nih.gov/pubmed/22574943
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.052
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