Allele-Selective Inhibition of Mutant Huntingtin Expression with Antisense Oligonucleotides Targeting the Expanded CAG Repeat

Huntington's disease (HD) is a currently incurable neurodegenerative disease caused by the expansion of a CAG trinucleotide repeat within the huntingtin (HTT) gene. Therapeutic approaches include selectively inhibiting the expression of the mutated HTT allele while conserving function of the no...

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Detalhes bibliográficos
Main Authors: Gagnon, Keith T., Pendergraff, Hannah M., Deleavey, Glen F., Swayze, Eric E., Potier, Pierre, Randolph, John, Roesch, Eric B., Chattopadhyaya, Jyoti, Damha, Masad J., Bennett, C. Frank, Montaillier, Christophe, Lemaitre, Marc, Corey, David R.
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2991413/
https://ncbi.nlm.nih.gov/pubmed/21028906
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/bi101208k
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