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.: US17014782Application Date: 2020-09-08
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Publication No.: US11110153B2Publication Date: 2021-09-07
- 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
- 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
- US20210008179A1 MODIFIED FACTOR IX, AND COMPOSITIONS, METHODS AND USES FOR GENE TRANSFER TO CELLS, ORGANS, AND TISSUES Public/Granted day:2021-01-14
Information query
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