UCL BUSINESS PLC;ST. JUDE CHILDREN'S RESEARCH HOSPITAL
发明人:
Nathwani Amit,McIntosh Jenny,Tuddenham Edward,Davidoff Andrew
申请号：
US201615254984
公开号：
US10124041(B2)
申请日:
2016.09.01
申请国别(地区):
美国
年份:
2018
代理人:
Marshall, Gerstein & Borun LLP
摘要：
There is provided a nucleic acid molecule comprising a nucleotide sequence encoding a factor VIII protein, wherein a B domain portion of the factor VIII protein is encoded by a nucleotides sequence between 90 and 111 nucleotides in length and has an amino acid sequence that is at least 85% identical to SEQ ID NO: 4 which comprises six asparagine residues. Also provided is a factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating Haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.
