Invention Grant
- Patent Title: Nucleic acid molecules encoding modified factor VIII proteins
- Patent Title (中): 编码修饰因子VIII蛋白的核酸分子
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Application No.: US14407008Application Date: 2013-06-12
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Publication No.: US09447168B2Publication Date: 2016-09-20
- Inventor: Amit Nathwani , Jenny McIntosh , Edward Tuddenham , Andrew Davidoff
- Applicant: UCL BUSINESS PLC , ST. JUDE CHILDREN'S RESEARCH HOSPITAL
- Applicant Address: GB London US TN Memphis
- Assignee: UCL BUSINESS PLC,ST. JUDE CHILDREN'S RESEARCH HOSPITAL
- Current Assignee: UCL BUSINESS PLC,ST. JUDE CHILDREN'S RESEARCH HOSPITAL
- Current Assignee Address: GB London US TN Memphis
- Agency: Marshall, Gerstein & Borun LLP
- Priority: GB1210357.8 20120612
- International Application: PCT/GB2013/051551 WO 20130612
- International Announcement: WO2013/186563 WO 20131219
- Main IPC: C12N15/52
- IPC: C12N15/52 ; C07K14/755 ; A01K67/027 ; A61K38/37

Abstract:
There is provided a nucleic acid molecule comprising a nucleotide sequence encoding for a functional factor VIII protein, wherein the portion of the nucleotide sequence encoding for the B domain of the factor VIII protein is between 90 and 111 nucleotides in length and encodes for an amino acid sequence comprising a sequence having at least 85% identity to SEQ ID NO: 4 and which comprises six asparagine residues. Also provided is a functional factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating hemophilia, e.g. hemophilia A, and a method for the preparation of a parvoviral gene delivery vector.
Public/Granted literature
- US20150158930A1 FACTOR VIII SEQUENCES Public/Granted day:2015-06-11
Information query
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