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Adeno-associated virus factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
专利权人:
BioMarin Pharmaceutical Inc.
发明人:
Stuart Bunting,Peter Cameron Colosi,Erno Pungor
申请号:
US15274046
公开号:
US10512675B2
申请日:
2016.09.23
申请国别(地区):
US
年份:
2019
代理人:
摘要:
The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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