1. From the Cardeza Foundation for Hematologic Research and Department of Medicine (L.S., Y.B., T.D.O., M.H., W.B.), Thomas Jefferson University, Philadelphia, PA; McAllister Heart Institute (L.S., Y.B., T.D.O., W.B.), and Department of Biochemistry and Biophysics (W.B.), University of North Carolina at Chapel Hill, Chapel Hill, NC; ExonHit Therapeutics (L.D., B.L.), Therapeutic Division, Paris, France; Portola Pharmaceuticals (P.A., P.B.C.), South San Francisco, CA.