An unbiased genome-wide analysis of zinc-finger nuclease specificity

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Richard Gabriel - , Deutsches Krebsforschungszentrum (DKFZ) (Autor:in)
  • Angelo Lombardo - , Vita-Salute San Raffaele University (Autor:in)
  • Anne Arens - , Deutsches Krebsforschungszentrum (DKFZ) (Autor:in)
  • Jeffrey C. Miller - , Sangamo BioSciences (Autor:in)
  • Pietro Genovese - , Vita-Salute San Raffaele University (Autor:in)
  • Christine Kaeppel - , Deutsches Krebsforschungszentrum (DKFZ) (Autor:in)
  • Ali Nowrouzi - , Deutsches Krebsforschungszentrum (DKFZ) (Autor:in)
  • Cynthia C. Bartholomae - , Deutsches Krebsforschungszentrum (DKFZ) (Autor:in)
  • Jianbin Wang - , Sangamo BioSciences (Autor:in)
  • Geoffrey Friedman - , Sangamo BioSciences (Autor:in)
  • Michael C. Holmes - , Sangamo BioSciences (Autor:in)
  • Philip D. Gregory - , Sangamo BioSciences (Autor:in)
  • Hanno Glimm - , Nationales Centrum für Tumorerkrankungen Dresden (NCT/UCC), Nationales Centrum für Tumorerkrankungen Dresden, Nationales Zentrum für Tumorerkrankungen (NCT) Heidelberg (Autor:in)
  • Manfred Schmidt - , Deutsches Krebsforschungszentrum (DKFZ) (Autor:in)
  • Luigi Naldini - , Vita-Salute San Raffaele University (Autor:in)
  • Christof Von Kalle - , Deutsches Krebsforschungszentrum (DKFZ) (Autor:in)

Abstract

Zinc-finger nucleases (ZFNs) allow gene editing in live cells by inducing a targeted DNA double-strand break (DSB) at a specific genomic locus. However, strategies for characterizing the genome-wide specificity of ZFNs remain limited. We show that nonhomologous end-joining captures integrase-defective lentiviral vectors at DSBs, tagging these transient events. Genome-wide integration site analysis mapped the actual in vivo cleavage activity of four ZFN pairs targeting CCR5 or IL2RG. Ranking loci with repeatedly detectable nuclease activity by deep-sequencing allowed us to monitor the degree of ZFN specificity in vivo at these positions. Cleavage required binding of ZFNs in specific spatial arrangements on DNA bearing high homology to the intended target site and only tolerated mismatches at individual positions of the ZFN binding sites. Whereas the consensus binding sequence derived in vivo closely matched that obtained in biochemical experiments, the ranking of in vivo cleavage sites could not be predicted in silico. Comprehensive mapping of ZFN activity in vivo will facilitate the broad application of these reagents in translational research.

Details

OriginalspracheEnglisch
Seiten (von - bis)816-823
Seitenumfang8
FachzeitschriftNature biotechnology
Jahrgang29
Ausgabenummer9
PublikationsstatusVeröffentlicht - Sept. 2011
Peer-Review-StatusJa

Externe IDs

PubMed 21822255