Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
AssigneeThe Children's Hospital of Philadelphia
InventorsKatherine A. High, Xavier Anguela
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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.
Claims28
A recombinant adeno-associated virus (rAAV) vector comprising a vector genome encapsidated by an AAV capsid, wherein said vector genome comprises an AAV inverted terminal repeat (ITR) and a nucleic add sequence encoding human Factor IX (FIX) protein, wherein said nucleic add sequence encoding human FIX protein is at least 90% identical to SEQ ID NO:10, has a reduced number of CpG di-nucleotides compared to wild-type nucleic add sequence encoding human FIX protein and encodes the same human FIX protein encoded by SEQ ID NO:
- 10.
Dependencies are inferred from the claim text.