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US 11,634,463 B2Granted
Methods and compositions for treating hemophilia
AssigneeSangamo Therapeutics, Inc.
InventorsJeffrey C. Miller, David Paschon, Edward J. Rebar
Life of this patent
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Abstract
Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias.
Claims11
A cell comprising a sequence encoding a Factor IX (F.IX) protein integrated into intron 1 of an endogenous albumin gene such that the F.IX protein is expressed from the albumin gene.
Dependencies are inferred from the claim text.