A GENE-THERAPY FOR DIABETES IN THE FUTURE

The concepts and methods of gene therapy are summarized in order to as sess a possible implication in the treatment of diabetes mellitus. Gen e therapy requires identification of the critical genetic defect and t hen the preparation and introduction of the therapeutic transgene, wit h an appropriate targeting and a strong regulated expression. The base s of the different human diabetic syndromes are reviewed in their pres ent state of knowledge : they are mostly clarified in the case of MODY , extreme insulin resistance syndromes, and some mitochondrial diabeti c syndromes; but still obscure in the case of Type 2 and Type 1 diabet ic syndromes. Substantial contributions to the understanding of the pa thophysiology of diabetes have been brought by transgenic animal model s. Gene therapy of human diabetic syndromes may become available, in a n undetermined future, particularly under the forms of insulin secreti ng transgenic ''organoids''. Such treatments should be proportionate t o the intrinsic severity of the candidate diseases and carefully scree ned for safety.