Inventors:
Philip D. GREGORY - Orinda CA, US
Katherine A. HIGH - Philadelphia PA, US
Michael C. HOLMES - Oakland CA, US
Hojun LI - Ellicott City MD, US
International Classification:
A61K 48/00, C12N 15/55, C12N 15/63, C12N 5/071, C12N 5/04, A61N 5/00, C12N 1/16, C12N 1/19, C12N 1/14, C12N 1/15, A61P 7/04, C12N 9/16, C12N 5/10
US Classification:
424 936, 435196, 536 232, 4353201, 424 946, 424 937, 514 44 R, 435366, 435325, 435410, 435419, 4352551, 4352542, 4352541, 43525411, 600 1
Abstract:
Disclosed herein are methods and compositions for insertion of Factor IX (FIX) sequences into the genome of a cell for treating hemophilia B.