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Related Experiment Video

Updated: Dec 18, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

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Lessons for the stem cell discourse from the gene therapy experience.

Theodore Friedmann1

  • 1Center for Molecular Genetics, Department of Pediatrics, UCSD School of Medicine, La Jolla, CA 92093-0634, USA. tfriedmann@ucsd.edu

Perspectives in Biology and Medicine
|October 18, 2005
PubMed
Summary

Human embryonic stem cell therapy holds promise for diseases but requires extensive research. Overstated expectations, similar to early gene therapy, risk damaging public trust and patient hope.

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Area of Science:

  • Regenerative Medicine
  • Stem Cell Biology
  • Medical Ethics

Background:

  • Human embryonic stem cells (hESCs) offer potential therapeutic applications for numerous diseases.
  • Early discussions of hESC therapies echo the exaggerated expectations seen in the initial phase of human gene therapy.
  • Hype in gene therapy previously damaged public perception and patient confidence.

Purpose of the Study:

  • To analyze the potential of hESCs in disease therapy.
  • To caution against unrealistic expectations and inaccurate representations in hESC therapeutic discussions.
  • To highlight the need for rigorous scientific and clinical research.

Main Methods:

  • Review of historical parallels with human gene therapy.
  • Analysis of current discourse surrounding hESC therapeutic applications.
Keywords:
Biomedical and Behavioral ResearchGenetics and Reproduction

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  • Assessment of the impact of public perception on emerging medical fields.

    • Main Results:

    • Exaggerated claims for hESC therapies parallel those in early gene therapy.
    • Unrealistic promises can lead to public distrust and disappointment, hindering progress.
    • Scientific and clinical validation are crucial for the successful translation of hESC therapies.

    Conclusions:

    • The therapeutic potential of hESCs is significant but requires substantial basic and preclinical research.
    • Managing public expectations and avoiding overpromising are critical to prevent setbacks.
    • Responsible communication and evidence-based advancement are essential for realizing hESC-based treatments.