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US 9,175,280 B2Granted
Methods and compositions for treating hemophilia B
AssigneesSangamo BioSciences, Inc., The Children's Hospital of Philadelphia
InventorsPhilip D. Gregory, Katherine A. High, Michael C. Holmes, Hojun Li
Life of this patent
Estimating the term…
Abstract
Disclosed herein are methods and compositions for insertion of Factor IX (FIX) sequences into the genome of a cell for treating hemophilia B.
Claims11
A protein comprising an engineered zinc finger protein DNA-binding domain that binds to a target site in an endogenous Factor IX (FIX) gene, wherein the DNA-binding domain comprises four or five zinc finger recognition regions ordered F1 to F4 or F1 to F5 from N-terminus to C-terminus, and wherein
- (i) when the DNA-binding domain comprises five zinc finger recognition regions, F1 to F5 comprise the following amino acid sequences:
- (SEQ ID NO:
- 4)
- F1:
- QSGDLTR
- (SEQ ID NO:
- 5)
- F2:
- RSDVLSE
- (SEQ ID NO:
- 6)
- F3:
- DRSNRIK
- (SEQ ID NO:
- 7)
- F4:
- RSDNLSE
- (SEQ ID NO:
- 8)
- F5:
- QNATRIN,
- wherein the DNA-binding domain binds to the target site shown in SEQ ID NO:3;
- and
- (ii) when the DNA-binding domain comprises four zinc finger recognition regions, F1 to F4 comprise the following amino acid sequences:
- (SEQ ID NO:
- 10)
- F1:
- RSDSLSV
- (SEQ ID NO:
- 11)
- F2:
- TSGHLSR
- (SEQ ID NO:
- 12)
- F3:
- RSDHLSQ
- (SEQ ID NO:
- 13)
- F4:
- HASTRHC,
- wherein the DNA-binding domain binds to the target site shown in SEQ ID NO:9.
38 more elements
Dependencies are inferred from the claim text.