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Updated: Sep 15, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
09:00

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

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Gene Editing Applications as Future Cardiovascular Therapies

Tomonori Tadokoro1,2, Eric N Olson1,2, Ning Liu1,2

  • 1Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, Texas, USA; email: Tomonori.Tadokoro@UTSouthwestern.edu, Eric.Olson@UTSouthwestern.edu, Ning.Liu@UTSouthwestern.edu.

Annual Review of Genetics
|July 16, 2025
PubMed

Insights

CRISPR-Cas9 gene editing shows promise for treating cardiovascular diseases by correcting genetic defects. Further research is needed to overcome delivery and safety challenges for clinical use.

Area of Science:

  • Biotechnology
  • Genetics
  • Cardiology

Background:

  • Cardiovascular disease is a major global health burden.
  • Current treatments have limitations.
  • CRISPR-Cas9 offers a novel therapeutic avenue.

Purpose of the Study:

  • To review gene editing strategies for cardiovascular disorders.
  • To discuss therapeutic applications, merits, and limitations.
  • To explore regulatory considerations for clinical translation.

Main Methods:

  • Review of current CRISPR-Cas9 gene editing technologies.
  • Analysis of potential applications in cardiovascular disease.
  • Evaluation of delivery methods and safety profiles.

Main Results:

  • CRISPR-Cas9 can correct genetic defects and modulate disease pathways.
  • In vivo delivery and safety are key challenges.
  • Significant therapeutic potential exists for various heart conditions.

Conclusions:

  • Gene editing holds transformative potential for cardiovascular medicine.
  • Addressing technical and safety hurdles is crucial for clinical adoption.
  • Continued advancements promise future therapeutic breakthroughs.

Keywords:
CRISPR-Cas9RNA editingadeno-associated viruscardiomyopathygene editingnanoparticle

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