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filingDate 2014-12-09-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2016-10-19-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber EP-3080143-A1
titleOfInvention Methods and compositions for treating hemophilia
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. In one aspect, described herein is a non-naturally occurring zinc-finger protein (ZFP) that binds to a target site in a region of interest (e.g., an albumin gene) in a genome, wherein the ZFP comprises one or more engineered zinc-finger binding domains. In certain embodiments, the zinc finger domain recognizes a target site in an albumin gene. In some embodiments, the zinc finger protein comprises five or six zinc finger domains designated and ordered F1 to F5 or F1 to F6, as shown in a single row of Table 1.
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