Safety and in vivo Expression of a GNE-Transgene: A Novel Treatment Approach for Hereditary Inclusion Body Myopathy-2

Hereditary inclusion body myopathy-2 (HIBM2) is an adult-onset, muscular disease caused by mutations in the GNE gene. HIBM2-associated GNE mutations causing hyposialyation have been proposed to contribute to reduced muscle function in patients with HIBM2, though the exact cause of this disease is un...

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Hlavní autoři: Phadke, Anagha P., Jay, Chris, Chen, Salina J., Haddock, Courtney, Wang, Zhaohui, Yu, Yang, Nemunaitis, Derek, Nemunaitis, Gregory, Templeton, Nancy S., Senzer, Neil, Maples, Phillip B., Tong, Alex W., Nemunaitis, John
Médium: Artigo
Jazyk:Inglês
Vydáno: Libertas Academica 2009
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Original Research
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2758285/
https://ncbi.nlm.nih.gov/pubmed/19838336
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