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Published on: September 2, 2021
Gene editing therapy as a therapeutic approach for cardiovascular diseases in animal models: A scoping review
Quan Duy Vo1,2
1Department of Cardiovascular Medicine, Faculty of Medicine, Dentistry and Pharmaceutical Sciences, Okayama University, Okayama Japan.
Insights
Gene editing therapies show promise for treating cardiovascular diseases (CVDs) by targeting genetic causes. Studies in animal models demonstrate high efficiency and safety, offering hope for future treatments.
Area of Science:
- Genetics
- Molecular Biology
- Cardiology
Background:
- Cardiovascular diseases (CVDs) are a leading global cause of death.
- Hereditary genetic factors significantly contribute to CVD burden.
- Current treatments manage symptoms but not underlying genetic causes.
Purpose of the Study:
- To review the efficacy and safety of gene editing interventions for CVDs.
- To explore advancements in gene therapy for cardiovascular conditions.
- To identify opportunities for future gene editing research in CVDs.
Main Methods:
- Systematic review of studies on gene editing in animal models of CVDs.
- Literature search conducted in PubMed, ScienceDirect, and Web of Science up to December 2024.
- Analysis of gene editing tools, targets, efficiency, and safety profiles.
Main Results:
- 57 studies included, predominantly using mouse models (86%).
- CRISPR-Cas9 (53%) and AAV vectors (80%) were common tools targeting genes like PCSK9 and LDLR.
- High editing efficiencies (25-85%) achieved; base editors showed better safety profiles than CRISPR-Cas9.
Conclusions:
- Gene editing therapy demonstrates significant potential for CVD treatment.
- High efficiency, therapeutic outcomes, and favorable safety observed in animal models.
- Further innovation and evaluation are crucial for clinical translation of gene editing for CVDs.
Background:
Cardiovascular diseases (CVDs) are the leading cause of mortality worldwide, with hereditary genetic factors contributing substantially to disease burden. Current treatments, including lifestyle modifications, pharmacotherapy, and surgical interventions, focus primarily on symptom management but fail to address underlying genetic causes, often resulting in disease progression or recurrence. Gene therapy has emerged as a transformative approach, offering a potential treatment. This review explores its efficacy and safety in animal models, identifying opportunities for future advancements.
Methods:
This review investigated studies on gene editing interventions in animal models of CVDs, retrieved from PubMed, ScienceDirect, and Web of Science up to December 2024.
Result:
A total of 57 studies were included in this review. Mice (86%) were the predominant model, with CRISPR-Cas9 (53%) and AAV vectors (80%) as the most used tools. Key targets included PCSK9 (32%), LDLR (9%), and MYH6/7 (7%), achieving 25-85% editing efficiency in liver/heart tissues. Base editors (ABE/CBE) showed superior safety, with <1% off-targets versus CRISPR-Cas9's 2-5 off-targets per guide. Reported toxicity risks included liver injury (AAVs, 23%) and transient cytokine elevation (LNPs, 14%).
Conclusion:
Gene editing therapy shows great potential for treating CVDs, with high efficiency, strong therapeutic outcomes, and favorable safety in animal models. Continued innovation and rigorous evaluation could transform cardiovascular treatment, benefiting patients with untreatable conditions.
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