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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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hPSC gene editing for cardiac disease therapy

Amina Saleem1, Muhammad Khawar Abbas2, Yongming Wang3,4

  • 1Beijing Laboratory for Cardiovascular Precision Medicine, MOE Key Laboratory of Medical Engineering for Cardiovascular Diseases, MOE Key Laboratory of Remodeling Related Cardiovascular Disease, Beijing Collaborative Innovation Center for Cardiovascular Disorders, Research Institute Building, Beijinj Anzhen Hospital, Capital Medical University, Room 319, 2 Anzhen Road, Chaoyang District, Beijing, Beijing, 100029, China.

Pflugers Archiv : European Journal of Physiology
|September 27, 2022
PubMed

Insights

Human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs) advance cardiovascular disease (CVD) research. Genome-editing technologies applied to hPSCs improve the study and potential therapies for cardiac conditions.

Area of Science:

  • Biomedical Research
  • Stem Cell Biology
  • Genetics

Background:

  • Cardiovascular diseases (CVDs) are a major global cause of death.
  • Studying CVD mechanisms is hindered by the limited availability of human cardiomyocytes with suitable genetic backgrounds.
  • Human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs) offer a valuable model for CVD research.

Purpose of the Study:

  • To review the application of genome-editing techniques in human pluripotent stem cells (hPSCs) for studying cardiovascular diseases.
  • To highlight recent advancements in genome editing for hPSCs and their impact on cardiac disease research and therapy.

Main Methods:

  • Utilizing genome-editing systems such as zinc finger nucleases, transcription activator-like effector nucleases, and CRISPR/Cas9 to modify hPSCs.
  • Employing hPSC-CMs as a model system for investigating disease mechanisms.
  • Focusing on advancements in genome editing efficiency within hPSCs.

Main Results:

  • Genome-editing technologies have been successfully applied to hPSCs for CVD research.
  • Specific genome-editing tools like CRISPR/Cas9 have shown significant promise.
  • These advancements enhance the efficiency of cell-based studies for cardiac diseases.

Conclusions:

  • Genome editing in hPSCs is a powerful tool for understanding cardiovascular diseases.
  • hPSC-CMs combined with genome editing accelerate the study of cardiac disease mechanisms.
  • This approach holds potential for developing novel therapies for cardiac conditions.

Keywords:
CRISPR/Cas9Cardiovascular diseaseGenome editingTALENZFNhiPSC

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