[Is a gene therapy for diabetic syndromes foreseeable?].
The concepts and methods of gene therapy are summarized in order to assess a possible implication in the treatment of diabetes mellitus. Gene therapy requires identification of the critical genetic defect and then the preparation and introduction of the therapeutic transgene, with an appropriate targeting and a strong regulated expression. The bases of the different human diabetic syndromes are reviewed in their present state of knowledge: they are mostly clarified in the case of MODY, extreme insulin resistance syndromes, and some mitochondrial diabetic syndromes; but still obscure in the case of Type 2 and Type 1 diabetic syndromes. Substantial contributions to the understanding of the pathophysiology of diabetes have been brought by transgenic animal models. Gene therapy of human diabetic syndromes may become available, in an undetermined future, particularly under the forms of insulin secreting transgenic "organoïds". Such treatments should be proportionate to the intrinsic severity of the candidate diseases and carefully screened for safety.
Assan
R
R
Service de Diabétologie, Hôpital Bichat, Paris.
Clauser
E
E
Larger
E
E
D000818:Animals; D030801:Animals, Genetically Modified; D003920:Diabetes Mellitus / Q000628:therapy*; D003921:Diabetes Mellitus, Experimental / Q000628:therapy*; D004195:Disease Models, Animal; D005544:Forecasting; D015316:Genetic Therapy / Q000379:methods / Q000639:trends*; D006801:Humans; D013577:Syndrome
Une thérapie génique des syndromes diabétiques est-elle envisageable?