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Updated: Sep 15, 2025
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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
Summary
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.
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