[Gene therapy: reality and expectations].
Chemical and pharmaceutical research have provided physicians with an array of drugs that have beneficial effects on a variety of diseases. Such drugs, however, mostly help in controlling the manifestations of the pathological condition but do not permanently modify the underlying cause. Hence the necessity of new forms of therapy that change drastically the current approach to medical treatment. Gene therapy, with its potential to correct the malfunctioning genes at the origin of variety of diseases, seems to fulfill the requirements of this therapeutic "revolution". The feasibility of such an approach is underscored by the improved knowledge of the molecular mechanism and/or gene defects at the origin of acquired diseases widely spread in the population, and of more rare congenital conditions. The technical advances in molecular biology and genetic engineering achieved in the last ten years, offer the tools necessary to implement such therapeutic interventions. Here we present the approaches currently employed for gene therapy in the context of recent clinical trials. The scientific, ethical and economical implications deriving from a prospective routine use of gene therapy in the clinical setting are discussed.
Mariani
L
L
Facoltà di Medicina, Dipartimento di Farmacologia, Chemioterapia e Tossicologia Medica, Università degli Studi di Milano.
D005260:Female; D030342:Genetic Diseases, Inborn / Q000235:genetics* / Q000628:therapy; D015316:Genetic Therapy; D006801:Humans; D008297:Male