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:

Katherine A. HIGH,Xavier ANGUELA

申请号：

US16707984

公开号：

US20200129598A1

申请日:

2019.12.09

申请国别(地区):

US

年份:

2020

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.

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