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[Gene therapy]
1Département de Pédiatrie & Unité, INSERM 429, Hôpital Necker-Enfants Malades, Paris, France.
Abstract:
This paper considers the prerequisites for gene therapy. This new field of therapeutics has not yet proved its efficacity nor its safety. Use, as a drug of DNA molecules which can modify host genome and environment as well requires specific prerequisites; among them, animal experimental models are essential despite their limitations. The different preclinical and clinical steps in the design of gene therapy studies are also discussed.