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FACTOR IX GENE THERAPY
专利权人:
UCL Business LTD
发明人:
Amit Nathwani,Jenny Mcintosh,Nishil Patel
申请号:
US16563193
公开号:
US20190388523A1
申请日:
2019.09.06
申请国别(地区):
US
年份:
2019
代理人:
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.
来源网站:
中国工程科技知识中心
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http://www.ckcest.cn/home/

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