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Raper, S.E., Chirmule, N., Lee, F.S., Wivel, N.A., Bagg, A., Gao, G.P., Wilson, J.M. and Batshaw, M.L. (2003) Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Molecular and Genetic Metabolism, 80, 148-158. doi:10.1016/j.ymgme.2003.08.016

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• TITLE: Vectors for gene therapy: A place for DNA transposon

  AUTHORS: Benoît Chénais

  KEYWORDS: Keywords: Transposable Element; Gene Delivery; Non-Viral Vectors; Piggybac; Sleeping Beauty

  JOURNAL NAME: Open Journal of Genetics, Vol.3 No.2A, June 26, 2013

  ABSTRACT: Gene therapy offers important perspectives in current and future medicine but suffers from imperfect vectors for the delivery of the therapeutic gene. Most preclinical and clinical trials have been based on the use of viral vectors, which have evident advantages but also some serious disadvantages. In the past decade the use of DNA transposon-based systems for gene delivery has emerged as a non-viral alternative. DNA transposon vector engineering remains largely in a preclinical phase but some interesting results have been obtained. This mini-review aims to provide the current state of the art on DNA transposon vectors used in a gene therapy perspective.