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

Hudecek M, Ivics Z (2018)

Publication Type: Journal article, Review article

Publication year: 2018

Journal

Current Opinion in Genetics & Development Elsevier

Book Volume: 52

Pages Range: 100-108

DOI: 10.1016/j.gde.2018.06.003

Abstract

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.

Involved external institutions

Universitätsklinikum Würzburg DE Germany (DE) Paul Ehrlich Institute / Paul-Ehrlich-Institut – Bundesinstitut für Impfstoffe und biomedizinische Arzneimittel (PEI) DE Germany (DE)

How to cite

APA:

Hudecek, M., & Ivics, Z. (2018). Non-viral therapeutic cell engineering with the Sleeping Beauty transposon system. Current Opinion in Genetics & Development, 52, 100-108. https://doi.org/10.1016/j.gde.2018.06.003

MLA:

Hudecek, Michael, and Zoltan Ivics. "Non-viral therapeutic cell engineering with the Sleeping Beauty transposon system." Current Opinion in Genetics & Development 52 (2018): 100-108.

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