Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity

Huntington's disease (HD) is a neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the huntingtin (HTT) gene. Disease pathogenesis derives, at least in part, from the long polyglutamine tract encoded by mutant HTT. Therefore, considerable effort has been dedicated to th...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Sun, Xin, Marque, Leonard O., Cordner, Zachary, Pruitt, Jennifer L., Bhat, Manik, Li, Pan P., Kannan, Geetha, Ladenheim, Ellen E., Moran, Timothy H., Margolis, Russell L., Rudnicki, Dobrila D.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2014
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Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4222366/
https://ncbi.nlm.nih.gov/pubmed/25035419
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddu349
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