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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Detalles Bibliográficos
Autores principales: 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
Formato: Texto
Lenguaje:English
Publicado: Libertas Academica 2009
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2758285/
https://www.ncbi.nlm.nih.gov/pubmed/19838336

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