UCL BUSINESS PLC ;St. Jude Children's Research Hospital
发明人:
Nathwani Amit,McIntosh Jenny,Tuddenham Edward,Davidoff Andrew
申请号：
US201816031503
公开号：
US2018311317(A1)
申请日:
2018.07.10
申请国别(地区):
美国
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.
