Thrombosis Research Institute;UCL Business PLC;St. Jude Children's Research Hospital
发明人:
Amit Nathwani,Natalie Ward,Adrian Thrasher,Edward Tuddenham,John McVey,John Gray,Andrew Davidoff
申请号：
US15651698
公开号：
US20180008726A1
申请日:
2017.07.17
申请国别(地区):
US
年份:
2018
代理人:
摘要：
An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes arc alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
