在大脑上腺核胆固醇缩的基因疗法后,血液癌症

Christine N Duncan¹, Jacob R Bledsoe¹, Bartosz Grzywacz¹+20

• ¹From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).