Virus-mediated gene delivery for human gene therapy

After over 20years from the first application of gene transfer in humans, gene therapy is now a mature discipline, which has progressively overcome several of the hurdles that prevented clinical success in the early stages of application. So far, the vast majority of gene therapy clinical trials hav...

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Veröffentlicht in:Journal of controlled release 2012-07, Vol.161 (2), p.377-388
Hauptverfasser: Giacca, Mauro, Zacchigna, Serena
Format: Artikel
Sprache:eng
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Zusammenfassung:After over 20years from the first application of gene transfer in humans, gene therapy is now a mature discipline, which has progressively overcome several of the hurdles that prevented clinical success in the early stages of application. So far, the vast majority of gene therapy clinical trials have exploited viral vectors as very efficient nucleic acid delivery vehicles both in vivo and ex vivo. Here we summarize the current status of viral gene transfer for clinical applications, with special emphasis on the molecular properties of the major classes of viral vectors and the information so far obtained from gene therapy clinical trials. [Display omitted]
ISSN:0168-3659
1873-4995
DOI:10.1016/j.jconrel.2012.04.008