In vivo gene therapy of type I diabetic mellitus using a cationic emulsion containing an Epstein Barr Virus (EBV) based plasmid vector

Hyuk Sang Yoo, Osam Mazda, Hyeon Yong Lee, Jin Chul Kim, Seok Min Kwon, Jung Eun Lee, Ick Chan Kwon, Hesson Jeong, Yu Seok Jeong, Seo Young Jeong

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13 Citations (Scopus)

Abstract

A cationic emulsion containing an insulin expression plasmid was prepared for the treatment of type 1 diabetic mellitus (DM) in vivo. A rat proinsulin-1 gene was inserted to EBV-based plasmid vectors containing CAG promoter. Cationic emulsion composed of DOTAP and squalene was complexed with the plasmid DNA. An intravenous injection of cationic emulsion containing proinsulin gene decreased blood glucose levels for 7 days within normal range. The cationic emulsion exerted more profound effect on blood glucose levels compared to naked DNA. RT-PCR results confirmed that the proinsulin was expressed in several organs containing liver, lung, spleen, and kidney. The refractory response was invoked by multiple injections of naked DNA or cationic emulsion/DNA complex, which was later proven to be an immune response against expressed proinsulin. Therefore, the cationic emulsion showed a promising result as a novel insulin gene therapy vehicle by decreasing blood glucose level for a month.

Original languageEnglish
Pages (from-to)139-144
Number of pages6
JournalJournal of Controlled Release
Volume112
Issue number1
DOIs
Publication statusPublished - 2006 May 1
Externally publishedYes

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Keywords

  • Cationic emulsion
  • Diabetic
  • Gene therapy
  • Insulin

ASJC Scopus subject areas

  • Pharmaceutical Science

Cite this

Yoo, H. S., Mazda, O., Lee, H. Y., Kim, J. C., Kwon, S. M., Lee, J. E., Kwon, I. C., Jeong, H., Jeong, Y. S., & Jeong, S. Y. (2006). In vivo gene therapy of type I diabetic mellitus using a cationic emulsion containing an Epstein Barr Virus (EBV) based plasmid vector. Journal of Controlled Release, 112(1), 139-144. https://doi.org/10.1016/j.jconrel.2006.01.019