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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Bibliografiset tiedot
Päätekijät: 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
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Mary Ann Liebert, Inc. 2011
Aiheet:
Research Articles
Linkit: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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