Glucose transporter-2 (GLUT2) promoter mediated transgenic insulin production reduces hyperglycemia in diabetic mice

FEBS Lett. 2005 Oct 24;579(25):5759-64. doi: 10.1016/j.febslet.2005.09.060. Epub 2005 Oct 5.

Abstract

Insulin production afforded by hepatic gene therapy (HGT) retains promise as a potential treatment for type 1 diabetes, but successful approaches have been limited. We employed a novel and previously untested promoter for this purpose, glucose transporter-2 (GLUT2) to drive insulin production via delivery by recombinant adeno-associated virus (rAAV). In vitro, the GLUT2 promoter was capable of robust glucose-responsive expression in transduced HepG2 human hepatoma cells. Therefore, rAAV constructs were designed to express the furin-cleavable human preproinsulin B10 gene, under the control of the murine GLUT2 promoter and packaged for delivery with rAAV expressing the type 5 capsid. Streptozotocin-induced diabetic mice were subjected to hepatic portal vein injection immediately followed by implantation of a sustained-release insulin pellet to allow time for transgenic expression. All mice injected with the rAAV5-GLUT2-fHPIB10 virus remained euglycemic for up to 35 days post-injection, with 50% euglycemic after 77 days post-injection. In contrast, mock-injected mice became hyperglycemic within 15 days post-injection following dissolution of the insulin pellet. Serum levels of both human insulin and C-peptide further confirmed successful transgenic delivery by the rAAV5-GLUT2-fHPIB10 virus. These findings indicate that the GLUT2 promoter may be a potential candidate for regulating transgenic insulin production for hepatic insulin gene therapy in the treatment of type I diabetes.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Blood Glucose / analysis
  • Dependovirus / genetics
  • Diabetes Mellitus, Experimental / complications
  • Diabetes Mellitus, Experimental / therapy*
  • Diabetes Mellitus, Type 1 / complications
  • Diabetes Mellitus, Type 1 / therapy*
  • Genetic Therapy*
  • Glucose / metabolism
  • Glucose / pharmacology
  • Glucose Transporter Type 2 / genetics*
  • Humans
  • Insulin / biosynthesis
  • Insulin / genetics*
  • Liver / metabolism
  • Mice
  • Promoter Regions, Genetic / drug effects
  • Promoter Regions, Genetic / genetics
  • Transgenes

Substances

  • Blood Glucose
  • Glucose Transporter Type 2
  • Insulin
  • Glucose