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[Gene transfer and gene therapy].

Y Merrouche1, V Combaret, S Négrier

  • 1Laboratoire d'immunologie, centre Léon-Bérard, Lyon, France.

Pediatrie
|January 1, 1992
PubMed
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Gene transfer technology shows promise for human gene therapy, with early trials demonstrating safety and feasibility. Further research is needed to improve gene transfer efficiency and target cell knowledge for treating genetic and non-genetic diseases.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Context:

  • Human gene therapy research has advanced significantly over the past decade.
  • Retroviral gene transduction is a key technology being explored.
  • Early clinical trials have been conducted for adenosine deaminase deficiency and metastatic melanoma.

Purpose:

  • To review the current state and future prospects of gene transfer technology in human gene therapy.
  • To highlight the feasibility and safety demonstrated in initial clinical applications.
  • To identify challenges and areas for future research and development.

Summary:

  • Gene transfer technology, particularly retroviral gene transduction, has entered human clinical trials for conditions like adenosine deaminase deficiency and metastatic melanoma, showing feasibility and safety.

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  • Significant challenges remain, including understanding gene function, enhancing gene transfer efficiency, achieving site-specific in vivo correction, and characterizing target cells like hematopoietic stem cells.
  • Future clinical applications are anticipated for genetic disorders such as immunodeficiency, thalassemia, and hemophilia, as well as non-genetic conditions through lymphokine gene therapy for cancer.

    • Impact:

    • Demonstrates the potential of gene transfer as a therapeutic modality for a range of diseases.
    • Identifies critical areas for scientific investigation to optimize gene therapy protocols.
    • Paves the way for developing novel treatments for debilitating genetic and non-genetic disorders.