In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Sharma, Rajiv; Anguela, Xavier M.; Doyon, Yannick; Wechsler, Thomas; DeKelver, Russell C.; Sproul, Scott; Paschon, David E.; Miller, Jeffrey C.; Davidson, Robert J.; Shivak, David; Zhou, Shangzhen; Rieders, Julianne; Gregory, Philip D.; Holmes, Michael C.; Rebar, Edward J.; High, Katherine A.

doi:10.1182/blood-2014-12-615492

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Key Points AAV- and ZFN-mediated targeting of the albumin locus corrects disease phenotype in mouse models of hemophilia A and B. Robust expression from the albumin locus provides a versatile platform for liver-directed protein replacement therapy.