New perspectives for gene therapy in endocrinology

Gene therapy for endocrine diseases represents an exciting new type of mole cular intervention that may be a curative one. Endocrine disorders that mig ht be treated by gene therapy include monogenic diseases, such as GH defici ency and hypothalamic diabetes insipidus, and multifactorial diseases, such as diabetes mellitus, obesity and cancer. Premises seem promising for endo crine tumours, but many combined approaches of cell and gene therapy are fo reseeable also for other endocrine disorders. This review outlines the prin ciples of gene therapy, describes the endocrine disorders that might take a dvantage of gene transfer approaches, as well as the gene therapy intervent ions that have already been attempted, their major limitations and the prob lems that remain to be solved.