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. 2015 Oct 14;9:355. doi: 10.3389/fnins.2015.00355

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Copyright © 2015 Nafissi and Foldvari.

This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

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Schematic representation of the non-viral transposon-mediated site-specific gene therapy. The non-viral transposon is used as a bi-component DNA vector system for delivery and insertion of therapeutic transgenes from a DNA vector into the target cell chromosome. This system is composed of either one plasmid (cis) or two plasmids (trans) contains the therapeutic gene of interest (GOI) (green arrow) and its expression cargo such as promoter (orange box) flanked between the two transposon Inverted Terminal Repeats (ITR) (red circles), and the transposase enzyme (blue arrow) expression cargo. (1) After delivery into the intended target cell and transportation into the nucleus, through a “cut and paste” transposase activity, (2) the transposon is excised from the transposon donor DNA vector, and (3) the therapeutic transgene is integrated at a chromosomal site.