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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Detalhes bibliográficos
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2011
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Research Articles
Acesso em linha: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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