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Updated: Jul 17, 2026

Large Animal Model for Evaluating the Efficacy of the Gene Therapy in Ischemic Heart
Published on: September 2, 2021
Gene therapy and stem cell therapy for cardiovascular diseases today: a model for translational research
1The Zena and Michael A Wiener Cardiovascular Institute and The Marie-Josee and Henry R Kravis Center for Cardiovascular Health, Mount Sinai School of Medicine, New York, NY 10029-6574, USA. valentin.fuster@mssm.edu
Abstract:
Clinical trials looking at ways to promote myocardial regeneration have reported that the administered therapies have either neutral effects or modest benefits of questionable impact. These somewhat disappointing results should emphasize the need for translational research, with bidirectional feedback between the basic research laboratory and the clinical arena. Such a translational pathway is illustrated by the quest to find an effective therapy for restenosis, which culminated in the development of sirolimus. At this point a move away from the bedside and a return to the bench seems necessary to better understand the mechanisms of action of progenitor cells and stimulating factors. Without such basic knowledge research might be prematurely discouraged and the opportunity to fully understand the true potential of cardiovascular regenerative therapy might be missed.
Insights
Clinical trials for myocardial regeneration show limited success, highlighting the need for translational research. Further basic science understanding of progenitor cells is crucial for advancing cardiovascular regenerative therapy.
Area of Science:
- Cardiovascular Research
- Regenerative Medicine
- Translational Science
Background:
- Clinical trials investigating myocardial regeneration therapies report neutral or minimally beneficial outcomes.
- Disappointing results necessitate a stronger connection between basic research and clinical applications.
Purpose of the Study:
- To emphasize the importance of translational research in cardiovascular regenerative therapy.
- To advocate for a return to basic science research to understand underlying mechanisms.
Main Methods:
- Review of clinical trial outcomes for myocardial regeneration.
- Analysis of the translational pathway exemplified by restenosis therapy development (sirolimus).
- Identification of knowledge gaps in progenitor cell and growth factor mechanisms.
Main Results:
- Current myocardial regeneration therapies offer limited clinical benefit.
- Translational research, like that for restenosis, can yield significant therapeutic advancements.
- A deeper understanding of cellular mechanisms is lacking.
Conclusions:
- Further basic research into progenitor cells and stimulating factors is essential.
- Without enhanced fundamental knowledge, cardiovascular regenerative therapy's potential may not be realized.
- A bidirectional approach between laboratory and clinic is vital for progress.
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