公开(公告)号：WO2016075473A3

公开(公告)日：2016-05-19

申请号：PCT/GB2015/053438

申请日：2015-11-12

申请人： UCL BUSINESS PLC

发明人： NATHWANI, Amit ,  MCINTOSH, Jenny ,  PATEL, Nishil

IPC分类号： C12N9/64

摘要： The invention relates to a new, more potent, coagulation factor IX (FIX) expression cassette for gene therapy of haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.