CRISPR Therapeutics AG
GENE EDITING FOR HEMOPHILIA A WITH IMPROVED FACTOR VIII EXPRESSION

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.