Non-viral therapeutic cell engineering with the Sleeping Beauty transposon system.

Widespread treatment of human diseases with gene therapies necessitates the development of gene transfer vectors that integrate genetic information effectively, safely and economically. Indeed, significant efforts have been devoted to engineer novel tools that (i) achieve high-level stable gene transfer at low toxicity to the host cell; (ii) induce low levels of genotoxicity and possess a `safe' integration profile with a high proportion of integrations into safe genomic locations; and (iii) are associated with acceptable cost per treatment, and scalable/exportable vector production to serve large numbers of patients. Two decades after the discovery of the Sleeping Beauty (SB) transposon, it has been transformed into a vector system that is fulfilling these requirements. Here we review recent developments in vectorization of SB as a tool for gene therapy, and highlight clinical development of the SB system towards hematopoietic stem cell gene therapy and cancer immunotherapy.
(Copyright © 2018 Elsevier Ltd. All rights reserved.)