Abstract | PURPOSE: To review the recent advances in the application of genetic modification strategies to the therapy of human diseases for which a molecular defect is known. METHODS: A computerized data bank search, the minutes of the National Institutes of Health (NIH) Recombinant DNA Advisory Committee published in the Federal Record, and reports of human clinical trials were used as data sources for this review. Clinical trials included in this review were published in the literature or approved by the NIH Recombinant DNA Advisory Committee. STUDY SELECTION: Evaluations of the efficacy of genetic modification strategies in clinical trials in human and in animal models are summarized. The design and outcome of the genetic modification strategies employed are reviewed for 16 marking trials, 16 gene replacement trials for molecular deficiency diseases, 3 chemoprotection and 4 chemotherapy sensitization trials, 11 cancer vaccine trials, 2 antisense oligonucleotide trials, and 3 molecular immunotherapy trials. DATA SYNTHESIS: CONCLUSIONS: The clinical application of genetic modification techniques has thus far been successful in the beginning phases of this field. These early results suggest that continuation of gene therapy trials designed to correct the molecular changes that lead to disease states in humans is warranted. Evaluation of such clinical trials in the future may be based on the analysis of assays for short-term surrogate endpoints, as well as on the therapeutic outcomes of the trial, such as survival or remission.
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Authors | E G Hanania, J Kavanagh, G Hortobagyi, R E Giles, R Champlin, A B Deisseroth |
Journal | The American journal of medicine
(Am J Med)
Vol. 99
Issue 5
Pg. 537-52
(Nov 1995)
ISSN: 0002-9343 [Print] United States |
PMID | 7485213
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S., Review)
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Topics |
- Bone Marrow Transplantation
- Clinical Trials as Topic
- Genetic Therapy
(methods)
- HIV Infections
(therapy)
- Humans
- Immunotherapy
- Neoplasms
(therapy)
- Treatment Outcome
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