Reversal of Diabetes Through Gene Therapy of Diabetic Rats by Hepatic Insulin Expression via Lentiviral Transduction

• DOI: 10.1038/mt.2012.8
• Publication Date: 2012-02-21T13:41:51Z
• Authors: Matthias Elsner; Taivankhuu Terbish; Anne Jörns; Ortwin Naujok; Dirk Wedekind; Hans-Jürgen Hedrich; Sigurd Lenzen
• Keywords: Proinsulin

Due to shortage of donor tissue a cure for type 1 diabetes by pancreas organ or islet transplantation is an option only very few patients. Gene therapy alternative approach the disease. Insulin generation in non-endocrine cells through genetic engineering promising therapeutic concept achieve insulin independence patients with diabetes. In present study furin-cleavable human was expressed liver autoimmune-diabetic IDDM rats (LEW.1AR1/Ztm-iddm) and streptozotocin-diabetic after portal vein injection INS-lentivirus. Within 5-7 days virus 7 × 10(9) INS-lentiviral particles blood glucose concentrations were normalized treated animals. This lowering effect remained stable year observation period. Human C-peptide as marker hepatic release range 50-100 pmol/ml serum. Immunofluorescence staining positive showing no signs transdifferentiation into pancreatic β-cells. shows that diabetic state can be efficiently reversed from somatic gene approach.