Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

A. Barzel, N. K. Paulk, Y. Shi, Y. Huang, K. Chu, F. Zhang, P. N. Valdmanis, L. P. Spector, M. H. Porteus, K. M. Gaensler and M. A. Kay ()
Additional contact information
A. Barzel: 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
N. K. Paulk: 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
Y. Shi: Box 1270, UCSF, San Francisco, California 94143-1270, USA
Y. Huang: 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
K. Chu: 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
F. Zhang: 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
P. N. Valdmanis: 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
L. P. Spector: 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
M. H. Porteus: 269 Campus Drive, Lorry Lokey Stem Cell Research Building, Room G3045, Stanford, California 94305-5164, USA
K. M. Gaensler: Box 1270, UCSF, San Francisco, California 94143-1270, USA
M. A. Kay: 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA

Nature, 2015, vol. 517, issue 7534, 360-364

Abstract: Promoterless recombinant adeno-associated virus is used without nucleases to target the human coagulation factor IX gene to the liver-expressed albumin locus in haemophilia B mice, with an on-target integration into ∼0.5% of the albumin alleles in hepatocytes; stable F9 plasma levels at 7–20% of normal were obtained, leading to normal coagulation times in treated factor-IX-deficient mice.

Date: 2015
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DOI: 10.1038/nature13864

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