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A Therapeutic Double Whammy: Transcriptional or Post-transcriptional Suppression of Microsatellite Repeat Toxicity by

Ashish N Rao1, Thomas A Cooper2

  • 1Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, TX 77030, USA; Department of Pathology and Immunology, Baylor College of Medicine, Houston, TX 77030, USA.

Molecular Cell
|November 4, 2017
PubMed

Insights

Microsatellite expansion diseases stem from unstable DNA repeats. Researchers are exploring CRISPR-based strategies, targeting either DNA or RNA, to reduce pathogenic repeat numbers and treat these conditions.

Area of Science:

  • Genetics
  • Molecular Biology
  • Biotechnology

Background:

  • Microsatellite expansion diseases arise from unstable tandem DNA repeats exceeding a pathogenic threshold.
  • These repeat expansions are implicated in various genetic disorders.

Purpose of the Study:

  • To explore novel therapeutic strategies for microsatellite expansion diseases.
  • To evaluate the potential of targeting repeat expansions using deactivated Cas9 (dCas9) systems.

Main Methods:

  • Two distinct approaches utilizing dCas9 were investigated.
  • One strategy targeted the DNA repeats directly.
  • The other strategy targeted the RNA transcripts of the repeats.

Main Results:

  • Both DNA-targeting and RNA-targeting dCas9 strategies demonstrated potential in reducing pathogenic repeat numbers.
  • These findings suggest distinct therapeutic avenues for microsatellite expansion disorders.

Conclusions:

  • Targeting either DNA or RNA repeats with dCas9 offers a promising therapeutic avenue for microsatellite expansion diseases.
  • Further research into these CRISPR-based interventions could lead to effective treatments.

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