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Gene therapy: current status and future prospects

W G Kerr1, J J Mulé

  • 1Department of Gene Therapy, SyStemix, Palo Alto, California.

Journal of Leukocyte Biology
|August 1, 1994
PubMed
Summary
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Gene therapy introduces foreign genes into cells for treating genetic, malignant, and infectious diseases. This review analyzes clinical trials assessing gene therapy

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Medical Genetics

Background:

  • Gene therapy offers a novel approach to treating human diseases by introducing foreign genes into eukaryotic cells.
  • It is being applied to genetic, malignant, and infectious diseases.

Purpose of the Study:

  • To review the current status of gene therapy by analyzing clinical protocols.
  • To evaluate the potential benefits of somatic cell-based gene therapies for various diseases.
  • To identify shortcomings and technical challenges for widespread gene therapy adoption.

Main Methods:

  • Analysis of clinical protocols for gene transfer into somatic cell targets.
  • Examination of trials involving marker or therapeutic gene(s).

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Main Results:

  • Gene therapy is entering the clinical arena with ongoing trials.
  • Trials are assessing gene therapy's efficacy alone or as an adjunct to conventional treatments.
  • The potential benefit for diseases unresponsive to conventional therapies is under investigation.

Conclusions:

  • Gene therapy holds promise for treating a range of human diseases.
  • Further research is needed to overcome technical hurdles for widespread clinical use.
  • Clinical trials are crucial for validating the efficacy and safety of gene therapy approaches.