Gene therapy and stem cell therapy for cardiovascular diseases today: a model for translational research

Valentin Fuster1, Javier Sanz

  • 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

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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