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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Detalhes bibliográficos
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
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2012
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Research Articles
Acesso em linha: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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