Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.

@article{Gagnon2010AlleleselectiveIO,
  title={Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.},
  author={Keith T Gagnon and Hannah M. Pendergraff and Glen F. Deleavey and Eric E. Swayze and Pierre Potier and John Randolph and Eric B Roesch and Jyoti Chattopadhyaya and Masad J Damha and C. F. Bennett and Christophe Montaillier and Marc Lemaitre and David R. Corey},
  journal={Biochemistry},
  year={2010},
  volume={49 47},
  pages={10166-78}
}
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 normal allele. We have evaluated a series of antisense oligonucleotides (ASOs) targeted to the expanded CAG repeat within HTT mRNA for their ability to selectively inhibit expression of mutant HTT protein… CONTINUE READING