UCL Business PLC;Thrombosis Research Institute;ST. JUDE CHILDREN'S RESEARCH HOSPITAL
发明人:
申请号：
EP13192973.9
公开号：
EP2698163A1
申请日:
2010.07.08
申请国别(地区):
EP
年份:
2014
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 are alpha- 1 -antitrypsin, as well as those involved in the coagulation cascade, hepatocyte 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.
