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Updated: Aug 10, 2026
Neo-Islet Formation in Liver of Diabetic Mice by Helper-dependent Adenoviral Vector-Mediated Gene Transfer
Published on: October 10, 2012
In vivo gene therapy of type I diabetic mellitus using a cationic emulsion containing an Epstein Barr Virus (EBV)
Hyuk Sang Yoo1, Osam Mazda, Hyeon Yong Lee
1Department of Biomaterials Engineering, School of Biotechnology and Bioengineering, Kangwon National University, Chunchon, 200-701 South Korea. hsyoo@kangwon.ac.kr
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.
