[Transgenesis and gene therapy].
There are now rather straightforward methods to create transgenic animals whose genome is altered at the germline level. One method consists in the micro-injection of a gene into the pronucleus of a fertilized egg, the second one involves an homologous recombination event obtained in embryonic stem cells in culture. Only the latter method could eventually lead to an authentic gene therapy since it could actually substitute a normal gene for a mutated one instead of merely introducing a supplementary gene as done by micro-injection. Description of these techniques makes it obvious that germline therapy of human beings would not only be inacceptable on ethical grounds but would also hardly have any medical indications. Quite on the contrary, somatic gene therapy does not suffer from the same reservations and has numerous potential applications to man. As a matter of fact, several protocols have already received approval and have reached the stage of clinical trials: for example SCID (severe combined immunodeficiency due to a mutated adenosine deaminase gene), AIDS as well as some forms of malignant tumors.
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Institut de Génétique et Biologie cellulaire CNRS, Montpellier.
D000163:Acquired Immunodeficiency Syndrome / Q000235:genetics / Q000628:therapy; D000818:Animals; D030801:Animals, Genetically Modified; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D009369:Neoplasms / Q000235:genetics / Q000628:therapy; D016511:Severe Combined Immunodeficiency / Q000628:therapy
Transgénèse et thérapie génique.