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Introduction <p>Gene therapy offers a novel approach to heart failure treatment by targeting underlying molecular mechanisms and potentially providing durable, disease-modifying effects. While early trials have shown promise, concerns remain regarding its long-term efficacy, safety, and clinical applicability.</p> Methodology <p>A systematic literature search was conducted across PubMed, Scopus, Web of Science, and Cochrane databases using predefined keywords and Boolean operators. Studies published between January 1, 2000, and August 31, 2025, were screened for eligibility. Only human clinical trials investigating gene therapy for heart failure were included. Data were extracted and synthesized systematically. PROSPERO 2026 CRD420261372758.</p> Results &amp; Conclusion <p>Eight studies were included. Five (62.5%) reported no serious adverse events, while three (37.5%) noted safety concerns such as mortality, atrial flutter, and hematoma. Four studies (50%) demonstrated improvements in cardiac function, while the remaining showed limited or no efficacy. Gene therapy in heart failure shows variable efficacy with some promising outcomes. Safety concerns and inconsistent results highlight the need for larger, multicenter trials with long-term follow-up. Ethical, cost, and translational challenges must also be addressed to support future clinical integration.</p>

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Gene Therapy in Heart Failure: A Systematic Review of Therapeutic Potential, Limitations, and Future Directions

  • 期刊论文

  • 发表日期：

    2026年7月16日

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  • Ikponmwosa Jude Ogieuhi,
  • Olufemi Akinmeji,
  • Victor Oluwatomiwa Ajekiigbe,
  • Kayode Ogunniyi,
  • Chukwuemeka Christian Aghasili,
  • Boluwaduro Abasiekem Adeyemi,
  • Andrew Ndakotsu

摘要

Introduction

Gene therapy offers a novel approach to heart failure treatment by targeting underlying molecular mechanisms and potentially providing durable, disease-modifying effects. While early trials have shown promise, concerns remain regarding its long-term efficacy, safety, and clinical applicability.

Methodology

A systematic literature search was conducted across PubMed, Scopus, Web of Science, and Cochrane databases using predefined keywords and Boolean operators. Studies published between January 1, 2000, and August 31, 2025, were screened for eligibility. Only human clinical trials investigating gene therapy for heart failure were included. Data were extracted and synthesized systematically. PROSPERO 2026 CRD420261372758.

Results & Conclusion

Eight studies were included. Five (62.5%) reported no serious adverse events, while three (37.5%) noted safety concerns such as mortality, atrial flutter, and hematoma. Four studies (50%) demonstrated improvements in cardiac function, while the remaining showed limited or no efficacy. Gene therapy in heart failure shows variable efficacy with some promising outcomes. Safety concerns and inconsistent results highlight the need for larger, multicenter trials with long-term follow-up. Ethical, cost, and translational challenges must also be addressed to support future clinical integration.