Virus-mediated gene delivery for human gene therapy

Mauro Giacca*, Serena Zacchigna

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

269 Citations (Scopus)

Abstract

After over 20 years 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.

Original languageEnglish
Pages (from-to)377-388
Number of pages12
JournalJournal of Controlled Release
Volume161
Issue number2
DOIs
Publication statusPublished - 20 Jul 2012

Keywords

  • Adeno-associated virus
  • Adenovirus
  • Gene therapy
  • Retrovirus
  • Viral vectors

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