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Are contemporary methods for somatic gene therapy suitable for clinical applications?
1Howard Hughes Medical Institute, Department of Cell Biology, Baylor College of Medicine, Houston, Texas.
Abstract:
Clinical trials involving the introduction of recombinant genes into human subjects began in 1989 after a decade of earnest debate concerning the technical, social, and ethical implications of somatic gene therapy. The initial trials involved the introduction of recombinant genes into peripheral blood lymphocytes to study the characteristics of tumor infiltrating lymphocytes, enhance immunotherapy for cancer, and treat severe combined immunodeficiency due to adenosine deaminase deficiency. Additional clinical trials involving the introduction of genes into bone marrow, hepatocytes, and tumor cells are underway. Are these clinical trials premature? This article reviews basic methods for somatic gene therapy and the clinical trials which have been proposed or performed to date. This clinical experience suggests that gene therapy can be performed in select clinical trials safely and with public acceptance. Clinical trials have provided essential data concerning the feasibility and safety of gene transfer in human subjects. These trials have also brought a clinical focus to the assessment of technologies currently being used in experimental models. While there will certainly be significant advances in somatic gene therapy in the future, existing methods may be employed fairly in clinical trials.
Insights
Somatic gene therapy clinical trials, initiated in 1989, have demonstrated safety and public acceptance. Early trials focused on lymphocytes for cancer and immunodeficiency, with ongoing studies in other tissues, showing feasibility for human gene transfer.
Area of Science:
- Biotechnology
- Medical Genetics
- Immunology
Background:
- Somatic gene therapy involves introducing recombinant genes into human cells.
- Ethical and technical debates preceded the first clinical trials in 1989.
- Initial trials targeted peripheral blood lymphocytes.
Purpose of the Study:
- Review basic methods for somatic gene therapy.
- Analyze proposed and performed clinical trials.
- Assess the safety and feasibility of gene transfer in humans.
Main Methods:
- Introduction of recombinant genes into peripheral blood lymphocytes.
- Gene transfer into bone marrow, hepatocytes, and tumor cells in ongoing trials.
- Review of existing clinical trial data.
Main Results:
- Gene therapy can be safely administered in select clinical trials.
- Public acceptance has been observed for gene therapy trials.
- Clinical trials provide essential data on gene transfer feasibility and safety.
Conclusions:
- Existing somatic gene therapy methods are suitable for current clinical trials.
- Clinical experience supports the safe application of gene therapy.
- Further advancements in somatic gene therapy are anticipated.