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Allele-Selective Inhibition of Mutant Huntingtin by Peptide Nucleic Acid (PNA)-Peptide Conjugates, Locked Nucleic Acid (LNA), and siRNA

The ability to inhibit expression of a mutant allele while retaining expression of wild-type protein might provide a useful approach to treating Huntington’s Disease (HD) and other inherited pathologies. The mutant form of huntingtin (HTT), the protein responsible for HD, is encoded by an mRNA conta...

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Autori principali: Hu, Jiaxin, Matsui, Masayuki, Corey, David R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2009
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3908448/
https://ncbi.nlm.nih.gov/pubmed/19796074
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/j.1749-6632.2009.04975.x
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