Gene targeting

Definition

Gene targeting is the process of altering a specific sequence or gene at its location in a genome. Potential modifications include deletion, insertion or replacement of endogenous sequence with alternative sequences. Targeting can be achieved by homologous recombination in some organisms (notably, mice), or with site-directed genome editing nucleases.

Latest Research and Reviews

  • Research |

    Genome editing in human zygotes shows that OCT4 is required for normal development at an earlier stage in humans than in mice.

    • Norah M. E. Fogarty
    • , Afshan McCarthy
    • , Kirsten E. Snijders
    • , Benjamin E. Powell
    • , Nada Kubikova
    • , Paul Blakeley
    • , Rebecca Lea
    • , Kay Elder
    • , Sissy E. Wamaitha
    • , Daesik Kim
    • , Valdone Maciulyte
    • , Jens Kleinjung
    • , Jin-Soo Kim
    • , Dagan Wells
    • , Ludovic Vallier
    • , Alessandro Bertero
    • , James M. A. Turner
    •  & Kathy K. Niakan
  • Research | | open

    CRISPR-Cas9-based gene editing involves double-strand breaks at target sequences, which are often repaired by mutagenic non-homologous end-joining. Here the authors use Cas9 nickases to generate coordinated single-strand breaks in donor and target DNA for precise homology-directed gene editing.

    • Xiaoyu Chen
    • , Josephine M. Janssen
    • , Jin Liu
    • , Ignazio Maggio
    • , Anke E. J. ‘t Jong
    • , Harald M.M. Mikkers
    •  & Manuel A. F. V. Gonçalves
  • Research | | open

    Understanding the link between epigenetic marks and gene regulation requires the development of new tools to directly manipulate chromatin. Here the authors demonstrate a Cas9-based system to recruit chromatin remodelers to loci of interest, allowing rapid, reversible manipulation of epigenetic states.

    • Simon M. G. Braun
    • , Jacob G. Kirkland
    • , Emma J. Chory
    • , Dylan Husmann
    • , Joseph P. Calarco
    •  & Gerald R. Crabtree
  • Research | | open

    β-thalassemia is characterised by the presence of an excess of α-globin chains, which contribute to erythrocyte pathology. Here the authors use CRISP/Cas9 to reduce α-globin expression in hematopoietic precursors, and show effectiveness in xenograft assays in mice.

    • Sachith Mettananda
    • , Chris A. Fisher
    • , Deborah Hay
    • , Mohsin Badat
    • , Lynn Quek
    • , Kevin Clark
    • , Philip Hublitz
    • , Damien Downes
    • , Jon Kerry
    • , Matthew Gosden
    • , Jelena Telenius
    • , Jackie A. Sloane-Stanley
    • , Paula Faustino
    • , Andreia Coelho
    • , Jessica Doondeea
    • , Batchimeg Usukhbayar
    • , Paul Sopp
    • , Jacqueline A. Sharpe
    • , Jim R. Hughes
    • , Paresh Vyas
    • , Richard J. Gibbons
    •  & Douglas R. Higgs
  • Reviews |

    Advances in genome sequencing, editing and synthetic biology have enhanced the feasibility of large-scale genome engineering, termed genome writing. In this Opinion article, Chari and Church discuss the strengths and limitations of diverse strategies for genome writing, including extensively modifying existing genomes versus synthesizing genomes de novo, and they provide future visions for writing large genomes.

    • Raj Chari
    •  & George M. Church

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