In vivo genome editing restores haemostasis in a mouse model of haemophilia

Hojun Li, Virginia Haurigot, Yannick Doyon, Tianjian Li, Sunnie Y. Wong, Anand S. Bhagwat, Nirav Malani, Xavier M. Anguela, Rajiv Sharma, Lacramiora Ivanciu, Samuel L. Murphy, Jonathan D. Finn, Fayaz R. Khazi, Shangzhen Zhou, David E. Paschon, Edward J. Rebar, Frederic D. Bushman, Philip D. Gregory, Michael C. Holmes () and Katherine A. High ()
Additional contact information
Hojun Li: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Virginia Haurigot: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Yannick Doyon: Sangamo BioSciences, Point Richmond Tech Center, 501 Canal Boulevard, Suite A100, Richmond, California 94804, USA
Tianjian Li: Sangamo BioSciences, Point Richmond Tech Center, 501 Canal Boulevard, Suite A100, Richmond, California 94804, USA
Sunnie Y. Wong: Sangamo BioSciences, Point Richmond Tech Center, 501 Canal Boulevard, Suite A100, Richmond, California 94804, USA
Anand S. Bhagwat: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Nirav Malani: 426 Johnson Pavilion, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, Pennsylvania 19104, USA
Xavier M. Anguela: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Rajiv Sharma: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Lacramiora Ivanciu: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Samuel L. Murphy: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Jonathan D. Finn: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Fayaz R. Khazi: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
Shangzhen Zhou: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard
David E. Paschon: Sangamo BioSciences, Point Richmond Tech Center, 501 Canal Boulevard, Suite A100, Richmond, California 94804, USA
Edward J. Rebar: Sangamo BioSciences, Point Richmond Tech Center, 501 Canal Boulevard, Suite A100, Richmond, California 94804, USA
Frederic D. Bushman: 426 Johnson Pavilion, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, Pennsylvania 19104, USA
Philip D. Gregory: Sangamo BioSciences, Point Richmond Tech Center, 501 Canal Boulevard, Suite A100, Richmond, California 94804, USA
Michael C. Holmes: Sangamo BioSciences, Point Richmond Tech Center, 501 Canal Boulevard, Suite A100, Richmond, California 94804, USA
Katherine A. High: CTRB 5000, Children’s Hospital of Philadelphia, 3501 Civic Center Boulevard

Nature, 2011, vol. 475, issue 7355, 217-221

Abstract: Gene correction in a blood disorder Direct editing of disease-causing mutations has obvious attractions for the treatment of genetic disorders if the many practical obstacles to the technique can be overcome. One promising line of research centres on the development of zinc finger nucleases (ZFNs) produced by fusing an engineered zinc finger DNA-binding domain to an endonuclease. These artificial enzymes induce efficient gene correction in cultured cells. Li et al. now report that zinc finger nucleases induce double-strand breaks in specifically selected locations on the genome and stimulate genome editing at a clinically meaningful level in vivo. In a proof-of-principle experiment, ZFNs delivered to the liver in a mouse model of haemophilia B achieved a level of gene replacement that was sufficient to correct the clotting defect, and the effect persisted following liver regeneration.

Date: 2011
References: Add references at CitEc
Citations: View citations in EconPapers (2)

Downloads: (external link)
https://www.nature.com/articles/nature10177 Abstract (text/html)
Access to the full text of the articles in this series is restricted.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:nat:nature:v:475:y:2011:i:7355:d:10.1038_nature10177

Ordering information: This journal article can be ordered from
https://www.nature.com/

DOI: 10.1038/nature10177

Access Statistics for this article

Nature is currently edited by Magdalena Skipper

More articles in Nature from Nature
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().