Send to

Choose Destination
See comment in PubMed Commons below
Curr Opin Immunol. 2012 Oct;24(5):609-16. doi: 10.1016/j.coi.2012.08.005. Epub 2012 Sep 13.

Development of nuclease-mediated site-specific genome modification.

Author information

  • 1Department of Pediatrics, Stanford University School of Medicine, Stanford, CA 94305, USA.


Genome engineering is an emerging strategy to treat monogenic diseases that relies on the use of engineered nucleases to correct mutations at the nucleotide level. Zinc finger nucleases can be designed to stimulate homologous recombination-mediated gene targeting at a variety of loci, including genes known to cause the primary immunodeficiencies (PIDs). Recently, these nucleases have been used to correct disease-causing mutations in human cells, as well as to create new animal models for human disease. Although a number of hurdles remain before they can be used clinically, engineered nucleases hold increasing promise as a therapeutic tool, particularly for the PIDs.

[PubMed - indexed for MEDLINE]
PubMed Commons home

PubMed Commons

How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for Elsevier Science
    Loading ...
    Support Center