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Applications of Sleeping Beauty transposons for nonviral gene therapy

Applications of Sleeping Beauty transposons for nonviral gene therapy Virus‐based gene therapy has advanced to clinical trials; however, this approach may result in serious adverse events including oncogenesis and the possibility of triggering fatal immune responses. Nonviral gene delivery approaches have a better safety profile, but their in vivo application has been largely limited in the past due to their inefficient delivery into cells and lack of stable chromosomal integration that is necessary for long‐term therapeutic benefit. However, recent advances suggest that the use of Sleeping Beauty transposons, a novel integrating nonviral vector system, are capable of achieving long‐lasting therapeutic levels of transgene expression in preclinical settings. These observations and other ongoing relevant studies may unlock the therapeutic potential of nonviral gene therapy for human diseases. iubmb Life, 59: 1 ‐ 6, 2007 http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png IUBMB Life Wiley

Applications of Sleeping Beauty transposons for nonviral gene therapy

IUBMB Life , Volume 59 (6) – Jan 1, 2007

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References (32)

Publisher
Wiley Subscription Services, Inc., A Wiley Company
Copyright
Copyright © 2007 International Union of Biochemistry and Molecular Biology
ISSN
1521-6543
eISSN
1521-6551
DOI
10.1080/15216540701435722
pmid
17613167
Publisher site
See Article on Publisher Site

Abstract

Virus‐based gene therapy has advanced to clinical trials; however, this approach may result in serious adverse events including oncogenesis and the possibility of triggering fatal immune responses. Nonviral gene delivery approaches have a better safety profile, but their in vivo application has been largely limited in the past due to their inefficient delivery into cells and lack of stable chromosomal integration that is necessary for long‐term therapeutic benefit. However, recent advances suggest that the use of Sleeping Beauty transposons, a novel integrating nonviral vector system, are capable of achieving long‐lasting therapeutic levels of transgene expression in preclinical settings. These observations and other ongoing relevant studies may unlock the therapeutic potential of nonviral gene therapy for human diseases. iubmb Life, 59: 1 ‐ 6, 2007

Journal

IUBMB LifeWiley

Published: Jan 1, 2007

Keywords: Nonviral gene therapy; transposase; transposon

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