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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...
Kaydedildi:
Asıl Yazarlar: | , , , , , , , , |
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Materyal Türü: | Artigo |
Dil: | Inglês |
Baskı/Yayın Bilgisi: |
2009
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Konular: | |
Online Erişim: | 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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