Vertex Pharmaceuticals Incorporated
COMPOSITIONS AND METHODS FOR EDITING BETA-GLOBIN FOR TREATMENT OF HEMAGLOBINOPATHIES

Abstract:

The disclosure features systems and methods for correcting a mutation in the human beta-globin (HBB) gene in a cell or population of cells. The disclosure also features methods of increasing repair of a DNA double stranded break (DSB) in an HBB gene by the homology-directed repair (HDR) pathway. The disclosure also features compositions for use in the methods.