Invention Grant
- Patent Title: Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
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Application No.: US15191357Application Date: 2016-06-23
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Publication No.: US10537617B2Publication Date: 2020-01-21
- Inventor: Katherine A. High , Xavier Anguela
- Applicant: The Children's Hospital of Philadelphia
- Applicant Address: US PA Philadelphia
- Assignee: THE CHILDREN'S HOSPITAL OF PHILADELPHIA
- Current Assignee: THE CHILDREN'S HOSPITAL OF PHILADELPHIA
- Current Assignee Address: US PA Philadelphia
- Agency: Pillsbury Winthrop Shaw Pittman LLP
- Agent Robert M. Bedgood
- Main IPC: A61K38/48
- IPC: A61K38/48 ; C12N15/86 ; C12N9/64

Abstract:
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.
Public/Granted literature
- US10799566B2 Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues Public/Granted day:2020-10-13
Information query
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