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A CRISPR-Cas13a Based Strategy That Tracks and Degrades Toxic RNA in Myotonic Dystrophy Type 1

Nan Zhang1, Brittani Bewick1, Guangbin Xia2

  • 1Department of Neurology, Houston Methodist Research Institute, Houston, TX, United States.

Frontiers in Genetics
|December 28, 2020
PubMed

Insights

This study shows CRISPR-Cas13a can target and reduce toxic RNA in myotonic dystrophy type 1. This RNA-targeting approach offers potential for treating genetic diseases caused by expanded repeat sequences.

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Myotonic dystrophy type 1 (DM1) is a neuromuscular disease caused by CTG repeat expansions in the DMPK gene.
  • Toxic RNA aggregates are a key driver of DM1 pathogenesis.
  • CRISPR-Cas13a systems offer RNA-targeting capabilities with therapeutic potential.

Purpose of the Study:

  • To investigate the use of Leptotrichia shahii (Lsh) Cas13a to target and eliminate toxic CUG repeat RNA in DM1.
  • To assess the therapeutic efficacy of LshCas13a in patient-derived myoblasts.
  • To explore the repurposing of deactivated LshCas13a for cellular RNA tracking.

Main Methods:

  • Biochemical assays to confirm CUG repeat RNA cleavage by LshCas13a.
  • Treatment of patient-derived myoblasts with LshCas13a and crRNA.
  • Analysis of splicing events and toxic RNA load in treated cells.
  • Utilizing deactivated LshCas13a for RNA-rich organelle visualization.

Main Results:

  • LshCas13a effectively cleaves CUG repeat RNA in vitro.
  • LshCas13a treatment significantly reduces toxic RNA load in DM1 patient myoblasts.
  • LshCas13a reverses DM1-associated missplicing events in key genes.
  • Deactivated LshCas13a successfully tracks RNA-rich organelles.

Conclusions:

  • LshCas13a demonstrates efficacy in targeting and reducing toxic RNA aggregates in DM1.
  • This study highlights the therapeutic potential of Cas13a for DM1 and other microsatellite expansion diseases.
  • The reprogrammability of LshCas13a offers versatile applications in RNA biology and disease treatment.
Keywords:
CRISPR-Cas13aRNA targetingmyotonic dystrophyneurodegenerationstress granule

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