preproinsulin gene therapy

Preproinsulin gene therapy is an experimental non-viral gene therapy designed for the treatment of type 1 diabetes. Developed by researchers at the Institute of Molecular Biology and Genetics and the Institute of Endocrinology and Metabolism in Kyiv, Ukraine, the therapy involves the delivery of a plasmid vector containing the human preproinsulin gene (HPG) to hepatocytes. The expression cassette is typically delivered using polyplexes (such as polyethylenimine-plasmid DNA complexes) or liposomes. Once delivered to the liver parenchyma, the transgene is expressed, leading to the synthesis and secretion of insulin and C-peptide, which helps normalize blood glucose levels and potentially reverse diabetes in animal models.

Other names

human preproinsulin gene therapynonviral human preproinsulin gene transferpreproinsulin gene transfer