Hereditary Inclusion Body Myopathy: Single Patient Response to Intravenous Dosing of GNE Gene Lipoplex

Hereditary inclusion body myopathy (HIBM) is an autosomal recessive adult-onset myopathy due to mutations in the GNE (UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase) gene. Affected patients have no therapeutic options. We have previously demonstrated in preclinical testing the abilit...

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Detaylı Bibliyografya
Asıl Yazarlar: Nemunaitis, Gregory, Jay, Chris M., Maples, Phillip B., Gahl, William A., Huizing, Marjan, Yardeni, Tal, Tong, Alex W., Phadke, Anagha P., Pappen, Beena O., Bedell, Cynthia, Allen, Henry, Hernandez, Cathy, Templeton, Nancy S., Kuhn, Joseph, Senzer, Neil, Nemunaitis, John
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Mary Ann Liebert, Inc. 2011
Konular:
Research Articles
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC3225042/
https://ncbi.nlm.nih.gov/pubmed/21517694
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2010.192
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