Clinical Considerations in the Design of Protocols for Somatic Gene Therapy
- 1 April 1991
- journal article
- research article
- Published by Mary Ann Liebert Inc in Human Gene Therapy
- Vol. 2 (1) , 77-83
- https://doi.org/10.1089/hum.1991.2.1-77
Abstract
Despite two decades of investigating the potential for somatic gene therapy in curing human disease, few clinical trials are being proposed. This delay is due in part to limitations of existing methods for gene transfer and to the recognized need to proceed judiciously into this controversial arena. Delay is also caused by a disjunction between the traditional precedents and principles of clinical investigation and the procedures instituted to regulate somatic gene therapy. The premise of this report is twofold: first, that more extensive clinical investigation of gene transfer technologies would be beneficial to patients, medicine, and basic science and second, that clinical investigations could be expedited by appealing to the established experience in clinical investigation without compromising the scientific excellence and discipline essential for this highly public process. This report develops a clinical perspective on potential applications of existing gene transfer technologies and the issues involved in developing experimental protocols. The initiation of clinical trials should be a primary goal of gene therapy research programs. The development of clinical trials involving somatic gene transfer requires consideration of a variety of clinical issues. The ethics and efficacy of somatic gene transfer trials may be largely dependent on the quality of patient selection and clinical follow-up. The success of these trials may be dependent not only on the sophistication of the molecular genetic technologies, but also on the adequacy of surgical, medical, and even psychosocial support for the patients. Optimal clinical trials involving gene transfer require a collaborative approach to these problems that draws from the experience of conventional clinical investigation as well as molecular genetics.Keywords
This publication has 21 references indexed in Scilit:
- In VivoExpression and Survival of Gene-Modified T Lymphocytes in Rhesus MonkeysHuman Gene Therapy, 1990
- Clinical application of somatic gene therapy in inborn errors of metabolismJournal of Inherited Metabolic Disease, 1990
- Human Gene Therapy, the Public, and Public PolicyHuman Gene Therapy, 1990
- The Role of Religions in the Analysis of the Ethical Issues of Human Gene TherapyHuman Gene Therapy, 1990
- Evolution of Ethical Debate about Human Gene TherapyHuman Gene Therapy, 1990
- The ethics of human gene therapyNature, 1986
- The Potentials of Inserting New Genetic InformationNew England Journal of Medicine, 1980
- Gene Therapy in Human Beings: When Is It Ethical to Begin?New England Journal of Medicine, 1980
- Gene Therapy for Human Genetic Disease?Science, 1972
- Will Society Be Prepared?Science, 1967