Allele-selective inhibition of trinucleotide repeat genes

Masayuki Matsui, David R. Corey

Research output: Contribution to journalReview articlepeer-review

27 Scopus citations


Expanded trinucleotide repeats cause Huntington's disease (HD) and many other neurodegenerative disorders. There are no cures for these devastating illnesses and treatments are urgently needed. Each trinucleotide repeat disorder is the result of the mutation of just one gene, and agents that block expression of the mutant gene offer a promising option for treatment. Therapies that block expression of both mutant and wild-type alleles can have adverse effects, challenging researchers to develop strategies to lower levels of mutant protein while leaving adequate wild-type protein levels. Here, we review approaches that use synthetic nucleic acids to inhibit expression of trinucleotide repeat genes.

Original languageEnglish (US)
Pages (from-to)443-450
Number of pages8
JournalDrug Discovery Today
Issue number9-10
StatePublished - May 2012

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery


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