title: [Gene therapy of the liver: from the laboratory to the patient's bedside].
abstract: Gene transfer techniques have significantly improved during the last few years. A number of therapeutic protocols has now been advanced for use in animal models and some are now in the first phases of clinical trials. In the field of hepatology, gene therapy represents a new therapeutic approach in the treatment of inherited hepatic diseases as well as liver tumors. In the present paper, we will review some examples of gene therapy of such diseases in animal models. Regarding inherited pathologies, Watanabe rabbits, which constitute a model of familial hypercholesterolemia, have been successfully treated by ex vivo gene transfer into hepatocytes using retroviral vectors. The genetic defect in Gunn rats, a model of type 1 Crigler-Najjar disease, has been corrected by in vivo retroviral mediated gene transfer. Adenoviral vectors have also been used to restore enzyme activity in OTC deficient mice. Concerning liver tumors, retroviral mediated transfer of a suicide gene into experimental liver metastasis has proven efficient in reducing the size of the tumors in treated animals. Taken together, these results clearly demonstrate the feasibility of treating hepatic diseases by gene transfer and may be considered as a promising therapeutic tool in hepatology.
authors.affiliation: Unité INSERM 370, CHU Necker Enfants Malades, Paris, France.
authors.forename: N
authors.initials: N
authors.lastname: Ferry
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000628:therapy; D008107:Liver Diseases / Q000628:therapy*; D008113:Liver Neoplasms / Q000628:therapy; D051379:Mice; D008815:Mice, Inbred Strains; D011817:Rabbits; D051381:Rats; D011911:Rats, Gunn
vernacular_title: Thérapie génique du foie: du laboratoire au chevet du malade.