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Gene therapy: here to stay

I D Dubé1, D Cournoyer

  • 1Gene Therapy Program (hematology/oncology), Toronto Hospital, Ont.

CMAJ : Canadian Medical Association Journal = Journal De L'Association Medicale Canadienne
|May 15, 1995
PubMed
Summary

Gene therapy, utilizing viral and nonviral methods for genetic modification, shows promise for treating diseases. Early clinical trials for conditions like adenosine deaminase deficiency and cancers indicate a future where gene therapy corrects genetic defects.

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Area of Science:

  • Biotechnology
  • Medical Research
  • Genetics

Background:

  • Gene therapy's feasibility was established using tumor viruses.
  • Viral and nonviral methods enable somatic cell genetic modification.
  • Two primary approaches: in-vivo and ex-vivo gene transfer.

Purpose of the Study:

  • To review the advancements and current status of gene therapy research.
  • To highlight the evolution of gene transfer techniques.
  • To assess the potential of gene therapy for various diseases.

Main Methods:

  • Development of viral and nonviral gene transfer vehicles.
  • Implementation of in-vivo and ex-vivo gene modification strategies.
  • Initiation and monitoring of clinical trials for genetic disorders and cancers.

Main Results:

  • First gene therapy clinical trial initiated in 1990 for adenosine deaminase deficiency.
  • Exponential increase in gene therapy clinical protocols worldwide.
  • Focus shifting towards common cancers and infectious diseases alongside rare metabolic disorders.

Conclusions:

  • Gene therapy is advancing rapidly, moving from rare diseases to common conditions.
  • Preliminary results support the potential of gene therapy to treat diseases by correcting genetic defects.
  • The goal of treating diseases by replacing or supplementing defective gene products is nearing reality.

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