Modified Factor IX, and compositions, methods and uses for gene transfer to cells, organs and tissues

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专利权人:

The Children's Hospital of Philadelphia

发明人:

High, Katherine A.,Anguela, Xavier

申请号：

AU2016282781

公开号：

AU2016282781A9

申请日:

2016.06.23

申请国别(地区):

AU

年份:

2019

代理人:

摘要：

The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses.In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/