Cells (Jan 2025)

CRISPR-Cas9 in Cardiovascular Medicine: Unlocking New Potential for Treatment

  • Klaudia Bonowicz,
  • Dominika Jerka,
  • Klaudia Piekarska,
  • Janet Olagbaju,
  • Laura Stapleton,
  • Munirat Shobowale,
  • Andrzej Bartosiński,
  • Magdalena Łapot,
  • Yidong Bai,
  • Maciej Gagat

DOI
https://doi.org/10.3390/cells14020131
Journal volume & issue
Vol. 14, no. 2
p. 131

Abstract

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Cardiovascular diseases (CVDs) remain a significant global health challenge, with many current treatments addressing symptoms rather than the genetic roots of these conditions. The advent of CRISPR-Cas9 technology has revolutionized genome editing, offering a transformative approach to targeting disease-causing mutations directly. This article examines the potential of CRISPR-Cas9 in the treatment of various CVDs, including atherosclerosis, arrhythmias, cardiomyopathies, hypertension, and Duchenne muscular dystrophy (DMD). The technology’s ability to correct single-gene mutations with high precision and efficiency positions it as a groundbreaking tool in cardiovascular therapy. Recent developments have extended the capabilities of CRISPR-Cas9 to include mitochondrial genome editing, a critical advancement for addressing mitochondrial dysfunctions often linked to cardiovascular disorders. Despite its promise, significant challenges remain, including off-target effects, ethical concerns, and limitations in delivery methods, which hinder its translation into clinical practice. This article also explores the ethical and regulatory considerations surrounding gene editing technologies, emphasizing the implications of somatic versus germline modifications. Future research efforts should aim to enhance the accuracy of CRISPR-Cas9, improve delivery systems for targeted tissues, and ensure the safety and efficacy of treatments in the long term. Overcoming these obstacles could enable CRISPR-Cas9 to not only treat but also potentially cure genetically driven cardiovascular diseases, heralding a new era in precision medicine for cardiovascular health.

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