Sleeping beauty transposon-mediated nonviral gene therapy.
Safe and effective delivery of genetic material to mammalian tissues would significantly expand the therapeutic possibilities for a large number of medical conditions. Unfortunately, the promise of gene therapy has been hampered by technical challenges, the induction of immune responses, and inadequate expression over time. Despite these setbacks, progress continues to be made and the anticipated benefits may come to fruition for certain disorders. In terms of delivery, nonviral vector systems are particularly attractive as they are simple to produce, can be stored for long periods of time, and induce no specific immune responses. A significant drawback to nonviral systems has been the lack of persistent expression, as plasmids are lost or degraded when delivered to living tissues. The recent application of integrating transposons to nonviral gene delivery has significantly helped to overcome this obstacle, because it allows for genomic integration and long-term expression. Recent advances in transposon-based vector systems hold promise as new technologies that may unlock the potential of gene therapy; however, technical and safety issues still need refinement.
Fernando
Stephen
S
Department of Pharmacology and Therapeutics, College of Medicine, University of Florida, Gainesville, Florida 32610-0267, USA.
Fletcher
Bradley S
BS
D000818:Animals; D004251:DNA Transposable Elements / Q000235:genetics*; D018390:Gene Targeting / Q000379:methods; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008954:Models, Biological; D012306:Risk; D019895:Transposases / Q000627:therapeutic use*
D004251:DNA Transposable Elements; D019895:Transposases; C494471:sleeping beauty transposase, human