Gene therapy for diabetes mellitus in rats by hepatic expression of insulin.


Journal Article

Type 1 diabetes mellitus is caused by severe insulin deficiency secondary to the autoimmune destruction of pancreatic beta cells. Patients need to be controlled by periodic insulin injections to prevent the development of ketoacidosis, which can be fatal. Sustained, low-level expression of the rat insulin 1 gene from the liver of severely diabetic rats was achieved by in vivo administration of a recombinant retroviral vector. Ketoacidosis was prevented and the treated animals exhibited normoglycemia during a 24-hr fast, with no evidence of hypoglycemia. Histopathological examination of the liver in the treated animals showed no apparent abnormalities. Thus, the liver is an excellent target organ for ectopic expression of the insulin gene as a potential treatment modality for type 1 diabetes mellitus by gene therapy.

Full Text

Cited Authors

  • Kolodka, TM; Finegold, M; Moss, L; Woo, SL

Published Date

  • April 1995

Published In

Volume / Issue

  • 92 / 8

Start / End Page

  • 3293 - 3297

PubMed ID

  • 7724555

Pubmed Central ID

  • 7724555

Electronic International Standard Serial Number (EISSN)

  • 1091-6490

International Standard Serial Number (ISSN)

  • 0027-8424

Digital Object Identifier (DOI)

  • 10.1073/pnas.92.8.3293


  • eng