[Gene therapy: a review 6 years after the first trials in man. Round Table No 6 at Giens XIII].
The first protocol of gene therapy was started in 1990 for two children with adenosine deaminase deficiency. In 1997, the field largely widened, with 2100 patients treated: 10 per cent suffer from hereditary disease, cystic fibrosis mainly, and 90 per cent acquired disease, mainly cancers or HIV infection. No toxicity of gene therapy has been observed for the patients or their environment. A psychological barrier has been crossed which might allow an easing of the regulations. Industrial firms are heavily engaged in this field and are defining the rules of production and use. After in vivo administration to patients, the gene is expressed but the vectorisation is still very weak. Gene therapy should thus be limited to indications with a well-established clinical and scientific rationale.
Favrot
M C
MC
Département de Biologie des Tumeurs, Centre Léon Bérard, Lyon, France.
D002648:Child; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D015316:Genetic Therapy; D015658:HIV Infections / Q000628:therapy; D006801:Humans; D009369:Neoplasms / Q000628:therapy; D016511:Severe Combined Immunodeficiency / Q000628:therapy