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the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine N.","family":"Duncan","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patricia L.","family":"Musolino","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Troy C.","family":"Lund","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashish O.","family":"Gupta","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Satiro","family":"De Oliveira","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian J.","family":"Thrasher","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Aubourg","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00f6rn-Sven","family":"K\u00fchl","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel J.","family":"Loes","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hernan","family":"Amartino","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicholas","family":"Smith","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juliana","family":"Folloni Fernandes","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Sevin","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raman","family":"Sankar","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shaun A.","family":"Hussain","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Gissen","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) \u2014 both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Universit\u00e9 Paris\u2013Saclay, H\u00f4pital Kremlin-Bic\u00eatre (P.A.), the Reference Center for Leukodystrophies, H\u00f4pital Kremlin-Bic\u00eatre, Assistance Publique\u2013H\u00f4pitaux de Paris, Universit\u00e9 Paris\u2013Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Universit\u00e9 de Pari..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Hugues","family":"Dalle","sequence":"additional","affiliation":[{"name":"From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana\u2013Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) \u2014 all in Massachusetts; 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