Gene editing for hemophilia A with improved factor VIII expression
AssigneesCrispr Therapeutics AG, Bayer HealthCare LLC
InventorAlan Richard Brooks
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Abstract
Provided herein, in some embodiments, are materials and methods for treating hemophilia A in a subject ex vivo or in vivo. Also provided herein, in some embodiments, are materials and methods for knocking in a coding sequence encoding a synthetic FVIII having a B domain substitute into a genome.
Claims13
A nucleic acid, comprising a nucleotide sequence encoding a synthetic FVIII protein, wherein the synthetic FVIII protein comprises a B domain substitute, and wherein the B domain substitute comprises the amino acid sequence of any one of SEQ ID NOs:
- 362-364, 366-369, 371, and 373.
A synthetic FVIII protein, wherein the synthetic FVIII protein comprises a B domain substitute, and wherein the B domain substitute comprises the amino acid sequence of any one of SEQ ID NOs:
- 362-364, 366-369, 371, and 373.
Dependencies are inferred from the claim text.