Methods and compositions for gene inactivation
AssigneeSangamo BioSciences, Inc.
InventorsDale Ando, Michael C. Holmes, Yann Jouvenot, Gary Ka Leong Lee
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Abstract
Disclosed herein are methods and compositions for inactivating CCR-5 genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs, such as adenovirus (Ad) vectors, and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided.
Claims13
A method for inactivating a CCR-5 gene in an isolated CD34+ cell, the method comprising administering to the cell a polynucleotide encoding a first fusion protein comprising a first cleavage domain and a first engineered zinc finger protein DNA-binding domain, wherein the DNA-binding domain comprises four zinc finger recognition regions ordered F1 to F4 from N-terminus to C-terminus, and wherein F1, F3, and F4 comprise the following amino acid sequences:
- F1:
- (SEQ ID NO:
- 2)
- DRSNLSR
- F3:
- (SEQ ID NO:
- 4)
- RSDNLAR
- F4:
- (SEQ ID NO:
- 8)
- TSGNLTR.
Dependencies are inferred from the claim text.