Targeting the Regulatory Genome to Treat Rare Blood Diseases
March 25, 2020
The gamma-globin gene is typically turned off at birth. By turning this single gene back on, we believe its possible to restore the body’s ability to make healthy red blood cells and provide a functional cure for sickle cell disease. Syros CMO David Roth, M.D., explains how in a conversation with Daniel Levine of RARECast