Gutless adenovirus: Last-generation adenovirus for gene therapy

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183 Citations (Scopus)

Abstract

Last-generation adenovirus vectors, also called helper-dependent or gutless adenovirus, are very attractive for gene therapy because the associated in vivo immune response is highly reduced compared to first- and second-generation adenovirus vectors, while maintaining high transduction efficiency and tropism. Nowadays, gutless adenovirus is administered in different organs, such as the liver, muscle or the central nervous system achieving high-level and long-term transgene expression in rodents and primates. However, as devoid of all viral coding regions, gutless vectors require viral proteins supplied in trans by a helper virus. To remove contamination by a helper virus from the final preparation, different systems based on the excision of the helper-packaging signal have been generated. Among them, Cre-loxP system is mostly used, although contamination levels still are 0.1–1% too high to be used in clinical trials. Recently developed strategies to avoid/reduce helper contamination were reviewed. © 2005 Nature Publishing Group. All rights reserved.
Original languageEnglish
Pages (from-to)S18-S27
JournalGene Therapy
Volume12
DOIs
Publication statusPublished - 1 Jan 2005

Keywords

  • Adenovirus
  • Gutless
  • Helper-dependent vectors
  • In vivo gene therapy

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