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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Mary Ann Liebert, Inc. 2012
Gaiak:
Research Articles
Sarrera elektronikoa: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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