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Gene Therapy for Cardiovascular Disease: Clinical Perspectives
Young Shin Lee1, Jung Ran Choi2, Jin-Bae Kim1,3
1Division of Cardiology, Department of Internal Medicine, College of Medicine, Kyung Hee University, Seoul, Korea.
Insights
Cardiac gene therapy shows promise for cardiovascular disease (CVD) but faces challenges like low efficiency and immune responses. Overcoming these hurdles is key to translating preclinical success into effective clinical treatments for CVD.
Area of Science:
- Cardiovascular Medicine
- Molecular Biology
- Biotechnology
Background:
- Cardiovascular disease (CVD) is a leading cause of death, with current treatments having limitations.
- Cardiac gene therapy offers a potential alternative for CVD management.
- Preclinical studies show promise, but clinical translation remains challenging.
Purpose of the Study:
- To review recent preclinical and clinical studies on cardiac gene therapy for CVD.
- To discuss the limitations and challenges hindering clinical application.
Main Methods:
- Comprehensive literature review of preclinical and clinical studies.
- Analysis of gene transduction efficiency, immune reactions, and off-target effects.
- Evaluation of successful and unsuccessful clinical trial outcomes.
Main Results:
- Significant success in animal models for various CVDs.
- Limited success in clinical applications, with few positive early trials.
- Persistent obstacles include low gene transduction efficiency and adverse immune responses.
Conclusions:
- Cardiac gene therapy holds potential but requires further research to overcome existing limitations.
- Addressing challenges in vector efficiency and immunogenicity is crucial for clinical success.
- Continued investigation into safe and effective gene delivery is essential for advancing CVD treatment.
Abstract:
Cardiovascular disease (CVD) stands as one of the leading causes of death in the United States, with its prevalence steadily on the rise. Traditional therapeutic approaches, such as pharmacological treatment, cardiovascular intervention, and surgery, have inherent limitations. In response to these challenges, cardiac gene therapy has emerged as a promising alternative for treating CVD patients. However, several obstacles persist, including the low efficiency of gene transduction, immune reactions to vectors or transduced cells, and the occurrence of off-target effects. While preclinical research has demonstrated significant success in various CVD model in both small and large animals, the translation of these findings to clinical applications has, for the most part, yielded disappointing results, except for some early, albeit small, trials. This review aims to provide a comprehensive summary of recent preclinical and clinical studies on gene therapy for various CVDs. Additionally, we discuss the existing limitations and challenges that hinder the widespread clinical application of cardiac gene therapy.
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