Inhibiting Expression of Mutant Huntingtin and Ataxin-3 by Targeting Expanded CAG Repeat RNAs

Many neurological disorders are caused by expanded trinucleotide repeats1, including Machado-Joseph Disease (MJD)2 and Huntington Disease (HD)3. MJD and HD are caused by expanded CAG repeats within the ataxin-3 (ATXN3) and huntingtin (HTT) genes. Inhibiting expression of ATXN3 or HTT are promising t...

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Hlavní autoři: Hu, Jiaxin, Matsui, Masayuki, Gagnon, Keith T., Schwartz, Jacob C., Gabillet, Sylvie, Arar, Khalil, Wu, Jun, Bezprozvanny, Ilya, Corey, David R.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2009
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2765218/
https://ncbi.nlm.nih.gov/pubmed/19412185
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nbt.1539
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