Gene therapy vectors: the prospects and potentials of the cut-and-paste transposons.
Gene therapy applications require efficient tools for the stable delivery of genetic information into eukaryotic genomes. Most current gene delivery strategies are based on viral vectors. However, a number of drawbacks, such as the limited cargo capacity, host immune response and mutational risks, highlight the need for alternative gene delivery tools. A comprehensive gene therapy tool kit should contain a range of vectors and techniques that can be adapted to different targets and purposes. Transposons provide a potentially powerful approach. However, transposons encompass a large number of different molecular mechanisms, some of which are better suited to gene delivery applications than others. Here, we consider the range and potentials of the various mechanisms, focusing on the cut-and-paste transposons as one of the more promising avenues towards gene therapy applications. Several cut-and-paste transposition systems are currently under development. We will first consider the mechanisms of piggyBac and the hAT family elements Tol1 and Tol2, before focusing on the mariner family elements including Mos1, Himar1 and Hsmar1.
Claeys Bouuaert
Corentin
C
School of Biomedical Sciences, University of Nottingham, Queen's Medical Centre, Nottingham NG7 2UH, UK.
Chalmers
Ronald M
RM
D000818:Animals; D002843:Chromatin / Q000235:genetics; D004247:DNA / Q000378:metabolism; D004251:DNA Transposable Elements; D018014:Gene Transfer Techniques; D005818:Genetic Engineering / Q000379:methods; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008957:Models, Genetic; D011995:Recombination, Genetic; D018626:Retroelements
D002843:Chromatin; D004251:DNA Transposable Elements; D018626:Retroelements; D004247:DNA