The Sleeping Beauty transposon system: a non-viral vector for gene therapy
Elena L Aronovich1, R Scott McIvor, Perry B Hackett
1Department of Genetics, Cell Biology and Development, The Center for Genome Engineering, Institute of Human Genetics, University of Minnesota, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA. arono001@umn.edu
Abstract:
Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading non-viral vector for gene therapy. This vector combines the advantages of viruses and naked DNA. Here we review progress over the last 2 years in vector design, methods of delivery and safety that have supported its use in the clinic. Currently, the SB vector has been validated for ex vivo gene delivery to stem cells, including T-cells for the treatment of lymphoma. Progress in delivery of SB transposons to liver for treatment of various systemic diseases, such as hemophilia and mucopolysaccharidoses types I and VII, has encountered some problems, but even here progress is being made.
More Related Videos
07:04Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System
Published on: February 4, 2021
10:58Transposon Mediated Integration of Plasmid DNA into the Subventricular Zone of Neonatal Mice to Generate Novel Models of Glioblastoma
Published on: February 22, 2015
Related Concept Videos
DNA-only Transposons
The donor site from where the transposon is excised is either degraded or...
Gene Therapy
