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CRISPR/Cas9-mediated genome editing in human stem cell-derived cardiomyocytes: Applications for cardiovascular

Effimia Christidi1, Haojun Margaret Huang1, Liam R Brunham2

  • 1Centre for Heart Lung Innovation, Department of Medicine, University of British Columbia, Vancouver, Canada.

Drug Discovery Today. Technologies
|September 13, 2018
PubMed

Insights

Human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs) and CRISPR/Cas9 genome editing offer powerful new ways to study cardiovascular diseases (CVDs) and drug-induced cardiotoxicity. These advanced models improve our understanding of disease mechanisms and potential treatments.

Area of Science:

  • Cardiology
  • Stem Cell Biology
  • Genetics

Background:

  • Cardiovascular diseases (CVDs) are a major global health concern.
  • Drug-induced cardiotoxicity frequently leads to medication withdrawal.
  • Current models for studying cardiac conditions require improvement.

Purpose of the Study:

  • To review advancements in using human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs) for studying CVDs.
  • To explore the application of CRISPR/Cas9 genome editing in modeling cardiotoxicity.
  • To highlight the potential of these technologies for investigating disease mechanisms and treatments.

Main Methods:

  • Utilizing human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs) as a model system.
  • Employing CRISPR/Cas9 genome editing for precise genetic manipulation.
  • Reviewing recent literature on the combined application of hPSC-CMs and CRISPR/Cas9.

Main Results:

  • hPSC-CMs provide a robust platform for studying cardiac function and disease.
  • CRISPR/Cas9 enables high-resolution investigation of genetic factors in cardiotoxicity.
  • The combination of these technologies facilitates the modeling of inherited and drug-induced cardiac conditions.

Conclusions:

  • hPSC-CMs and CRISPR/Cas9 significantly advance the study of cardiovascular diseases.
  • These tools offer new avenues for understanding drug-induced cardiotoxicity mechanisms.
  • Future research can leverage these models for novel therapeutic development.

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