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Human gene therapy

J S Sandhu1, A Keating, N Hozumi

  • 1Department of Surgery, University of Toronto, Canada.

Critical Reviews in Biotechnology
|January 1, 1997
PubMed
Summary
This summary is machine-generated.

Human gene therapy offers potential for treating genetic disorders like cystic fibrosis and cancer by inserting functional genes into cells. Ongoing research aims to overcome technical and ethical challenges for broader clinical application.

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Area of Science:

  • Biotechnology
  • Genetics
  • Medical Research

Background:

  • Gene therapy involves correcting genetic errors or adding new functions to human cells.
  • It holds promise for treating genetic disorders such as cystic fibrosis and cancer.

Purpose of the Study:

  • To discuss human gene therapy and its applications.
  • To explore methods and challenges in gene transfer for therapeutic purposes.

Main Methods:

  • Development of retroviral and non-viral vectors for ex vivo and in vivo gene transfer.
  • Engineering promoter systems for regulated gene expression in host cells.

Main Results:

  • Gene transfer techniques are utilized in basic research, including cancer studies.

Related Experiment Videos

  • Current efforts focus on gene insertion into somatic cells.

    • Conclusions:

    • Gene therapy has significant potential for treating genetic disorders.
    • Technical and ethical hurdles must be addressed for routine clinical use.
    • The human genome project aids in identifying disease-related genes.