UCL BUSINESS PLC;St. Jude Children's Research Hospital
发明人:
Amit Nathwani,Jenny McIntosh,Edward Tuddenham,Andrew Davidoff
申请号：
US16031503
公开号：
US20180311317A1
申请日:
2018.07.10
申请国别(地区):
US
年份:
2018
代理人:
摘要：
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 haemophilia, e.g. haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.
