Methods and compositions for treating hemophilia
First Claim
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1. A method of integrating a donor sequence encoding a Factor IX (F.IX) protein into an endogenous albumin gene in a cell, the method comprising:
- cleaving the endogenous albumin gene in the cell using at least one zinc finger nuclease (ZFN), at least one TAL-effector domain nuclease (TALEN) or at least one CRISPR/Cas nuclease system that binds to a target site in the endogenous albumin gene comprising at least 15 base pairs of SEQ ID NO;
2 or SEQ ID NO;
3 in the presence of the donor sequence such that the donor sequence is integrated into the endogenous albumin gene, the F.IX protein is expressed and secreted from the cell.
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Abstract
Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias.
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11 Claims
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1. A method of integrating a donor sequence encoding a Factor IX (F.IX) protein into an endogenous albumin gene in a cell, the method comprising:
cleaving the endogenous albumin gene in the cell using at least one zinc finger nuclease (ZFN), at least one TAL-effector domain nuclease (TALEN) or at least one CRISPR/Cas nuclease system that binds to a target site in the endogenous albumin gene comprising at least 15 base pairs of SEQ ID NO;
2 or SEQ ID NO;
3 in the presence of the donor sequence such that the donor sequence is integrated into the endogenous albumin gene, the F.IX protein is expressed and secreted from the cell.- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11)
Specification