Constitutive and regulated expression of processed insulin following in vivo hepatic gene transfer

Gene Ther. 2002 Jul;9(14):963-71. doi: 10.1038/sj.gt.3301746.

Abstract

To test whether hepatocytes engineered in vivo can serve as surrogate beta cells by similarly secreting mature insulin in a glucose-sensitive manner, we prepared adenoviral vectors encoding wild-type proinsulin (hIns-wt), a modified proinsulin cleavable by the ubiquitously expressed protease furin (hIns-M3), or each of the two beta cell specific pro-insulin convertases PC2 and PC3. Following a detailed in vitro characterization of the proteins produced by our vectors, we infected the liver and, for comparison, the muscle of a chemically induced murine model of type I diabetes. Insulin expression from the transduced tissues was extensively characterized and showed to be constitutive rather than regulated. To obtain regulated expression, we placed expression of hIns-M3 under the control of the dimerizer-inducible transcription system. Hormone secretion from mouse liver was negligible in the absence of the dimerizer drug rapamycin, was inducible in a dose-dependent manner upon its administration, and reversible following drug withdrawal. These data confirm liver as a promising target for in vivo expression of processed insulin. While suggesting that hepatocytes cannot provide authentic glucose-responsive regulation, these results demonstrate that pharmacological regulation is a promising alternative route to the controlled delivery of insulin following hepatic gene transfer.

Publication types

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

MeSH terms

  • Adenoviridae
  • Animals
  • Cells, Cultured
  • Combined Modality Therapy
  • Diabetes Mellitus, Experimental
  • Diabetes Mellitus, Type 1 / therapy*
  • Dimerization
  • Female
  • Gene Expression
  • Genetic Engineering / methods
  • Genetic Therapy / methods*
  • Genetic Vectors / pharmacology
  • Hepatocytes / drug effects
  • Hepatocytes / metabolism*
  • Insulin / metabolism
  • Insulin Secretion
  • Mice
  • Mice, Nude
  • Proinsulin / genetics*
  • Sirolimus / therapeutic use*
  • Transfection / methods

Substances

  • Insulin
  • Proinsulin
  • Sirolimus