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Gene therapy, concepts, current trials and future directions
1Research and Development, Genetic Therapy, Inc., Gaithersburg, Maryland 20878.
Annual Review of Pharmacology and Toxicology
|January 1, 1993
Summary
Gene therapy shows early promise for genetic diseases like ADA deficiency and cancers using retroviral vectors. Further research is needed for effective in vivo gene delivery and broader clinical applications.
Area of Science:
- Biotechnology
- Molecular Biology
- Medical Genetics
Background:
- Retroviral-mediated gene transfer clinical trials began in the USA in 1989.
- Gene therapy is being explored for treating genetic diseases and cancers.
- Gene marking is utilized in autologous bone marrow transplantation protocols.
Purpose of the Study:
- To review the progress and applications of gene therapy since its inception.
- To highlight the therapeutic potential and ongoing research in gene therapy.
- To discuss the development of vector systems and delivery methods.
Main Methods:
- Review of clinical trial data and proposed protocols.
- Analysis of gene-marking applications in cancer therapy.
- Evaluation of drug delivery potential using lymphokines and cytokines.
Main Results:
- Encouraging clinical benefits observed in ADA deficiency treatment.
- Gene marking extensively used in cancer and bone marrow transplantation.
- Testing initiated for Familial Hypercholesterolemia and Hemophilia B.
Conclusions:
- Gene therapy demonstrates early clinical success and rapid development.
- Vector systems and delivery methods are advancing, including liposome-based trials.
- Innovative research for in vivo vectors is crucial for realizing gene therapy's full potential.