Vertex Pharmaceuticals Incorporated
MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES

Abstract:

The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34.sup.+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and .beta.-thalassemia.