Gene therapy: too much splice can spoil the dish

J Clin Invest. 2012 May;122(5):1600-2. doi: 10.1172/JCI63066. Epub 2012 Apr 23.

Abstract

The use of integrating vectors for gene therapy - required for stable correction of gene expression - carries the risk of insertional mutagenesis, which can lead to activation of a tumorigenic program. In this issue of the JCI, Moiani et al. and Cesana et al. investigate how viral vectors can induce aberrant splicing, resulting in chimeric cellular-viral transcripts. The finding that this is a general phenomenon is concerning, but some of their results do suggest approaches for the development of safeguards in gene therapy vector design.

Publication types

  • Research Support, Non-U.S. Gov't
  • Comment

MeSH terms

  • Alternative Splicing*
  • Genome, Human*
  • Humans
  • Lentivirus / physiology*
  • Lentivirus Infections / genetics*
  • Protein Isoforms / genetics*
  • Transcriptome*
  • Virus Integration*

Substances

  • Protein Isoforms