Gene therapy for heart failure: A novel treatment for the age old disease

Neel Vora1, Parth Patel2, Aarsh Gajjar3

  • 1B. J. Medical College, Ahmedabad, India.

Disease-A-Month : DM
|September 21, 2023
PubMed

Insights

Gene therapy offers a promising new approach to treating heart failure, a leading cause of death. While challenges remain, research shows potential for future clinical applications in cardiovascular disease management.

Area of Science:

  • Cardiovascular Medicine
  • Molecular Biology
  • Regenerative Medicine

Background:

  • Cardiovascular disease (CVD) remains the primary global cause of mortality, with heart failure affecting millions.
  • Current heart failure treatments can only delay disease progression, highlighting the need for novel therapeutic strategies.
  • Gene therapy presents a potential breakthrough for addressing the limitations of existing heart failure care.

Approach:

  • This paper reviews the current landscape of gene therapy for heart failure, examining advances in delivery methods, therapeutic targets, and applications.
  • Literature search focused on preclinical and clinical studies, evaluating various gene therapy strategies and their efficacy.
  • Analysis includes methods like arterial/venous infusion, myocardial injection, and pericardial delivery, alongside specific gene targets.

Key Points:

  • Promising preclinical results observed with strategies such as AC6 expression, S100A1 upregulation, and VEGF-B/SDF-1 gene therapy.
  • Stimulating cardiomyocyte proliferation via cyclin A2 overexpression is a potential avenue for heart regeneration.
  • Delivery methods encompass diverse routes, including direct injection and systemic infusion, each with unique challenges and benefits.

Conclusions:

  • Significant obstacles persist, including definitive gene selection, optimized delivery systems, and robust preclinical/clinical trial methodologies.
  • Despite challenges, gene therapy for heart failure shows encouraging potential as a future treatment modality.
  • The feasibility and timeline for integrating gene therapy into standard cardiovascular care require further investigation and development.

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