Trinucleotide Repeat Expansion Diseases, RNAi, and Cancer

Andrea E. Murmann, Jindan Yu, Puneet Opal, Marcus E. Peter*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

18 Scopus citations


Many neurodegenerative diseases are caused by unstable trinucleotide repeat (TNR) expansions located in disease-associated genes. siRNAs based on CAG repeat expansions effectively kill cancer cell lines in vitro through RNAi. They also cause significant reduction in tumor growth in a human ovarian cancer mouse model with no toxicity to the treated mice. This suggests that cancer cells are particularly sensitive to CAG TNR-derived siRNAs, and explains a reported inverse correlation between the length of CAG TNRs and reduced global cancer incidences in some CAG TNR diseases. This review discusses both mutant proteins and mutant RNAs as a cause of TNR diseases, with a focus on RNAi and its role in contributing to disease pathology and in suppressing cancer.

Original languageEnglish (US)
Pages (from-to)684-700
Number of pages17
JournalTrends in Cancer
Issue number10
StatePublished - Oct 2018


  • AR
  • Huntington's disease
  • RNA
  • SBMA
  • TNR
  • cancer

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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