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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發表在: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.
格式: Artigo
語言:Inglês
出版: Oxford University Press 2014
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在線閱讀: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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