title: Gene and cell-based therapy for diabetes mellitus: endocrine gene therapeutics.
abstract: Recently, islet transplantation in the treatment of type 1 diabetes has been revisited with improved results. This approach has the potential to restore the regulatory unit of endocrine pancreas, but it cannot be a definite solution because of its limitation for the use of toxic immune-suppressive agents and limited number of donors. One possible way to restore the insulin secretion safely is with cell therapy using tissue engineering of a patient's own somatic cells by transduction of the corresponding gene of interest, in which extrapancreatic cells are engineered to secrete insulin. We constructed a somatic cell therapy system using a furin-cleavable insulin gene and somatic cells of mesenchymal origin. We also tested a semipermeable chamber that can contain allo- or xenogeneic insulin-secreting cells and found that the device could be helpful for cell therapy. This novel approach should provide a feasible method for treatment of diabetes mellitus without the use of any toxic agent or of embryonic stem cells that involve ethical implications.
authors.affiliation: Division of Diabetes, Metabolism and Endocrinology, Department of Internal Medicine, Institute of DNA Medicine, Jikei University School of Medicine, 3-25-8, Nishishinbashi, Minato-ku, Tokyo 105-8461, Japan. stakashi@jikei.ac.jp
authors.forename: Takashi
authors.initials: T
authors.lastname: Sasaki
authors.forename: Kei
authors.initials: K
authors.lastname: Fujimoto
authors.forename: Kyoko
authors.initials: K
authors.lastname: Sakai
authors.forename: Masami
authors.initials: M
authors.lastname: Nemoto
authors.forename: Nozomu
authors.initials: N
authors.lastname: Nakai
authors.forename: Naoko
authors.initials: N
authors.lastname: Tajima
mesh_terms: D003922:Diabetes Mellitus, Type 1 / Q000628:therapy*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000295:instrumentation / Q000379:methods*; D006801:Humans; D016381:Islets of Langerhans Transplantation; D023822:Tissue Engineering / Q000295:instrumentation / Q000379:methods*