Safe Harbours for the Integration of New DNA in the Human Genome

Nat Rev Cancer. 2011 Dec 1;12(1):51-8. doi: 10.1038/nrc3179.

Abstract

Interactions between newly integrated DNA and the host genome limit the reliability and safety of transgene integration for therapeutic cell engineering and other applications. Although targeted gene delivery has made considerable progress, the question of where to insert foreign sequences in the human genome to maximize safety and efficacy has received little attention. In this Opinion article, we discuss 'genomic safe harbours' - chromosomal locations where therapeutic transgenes can integrate and function in a predictable manner without perturbing endogenous gene activity and promoting cancer.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cell Engineering
  • Dependovirus / genetics
  • Gene Transfer Techniques
  • Genetic Therapy*
  • Genome, Human*
  • Humans
  • Mice
  • Neoplasms / genetics
  • Neoplasms / therapy*
  • Proteins / genetics
  • RNA, Untranslated
  • Receptors, CCR5 / genetics
  • Virus Integration*

Substances

  • Gt(ROSA)26Sor non-coding RNA, mouse
  • Proteins
  • RNA, Untranslated
  • Receptors, CCR5