AustralieFrV1, Main, Exploration, bibRecord, 000B00

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

Identifieur interne : 000B00 ( Main/Exploration ); précédent : 000A99; suivant : 000B01

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

Auteurs : Florian Eichler ; Christine Duncan ; Patricia L. Musolino ; Paul J. Orchard ; Satiro De Oliveira ; Adrian J. Thrasher ; Myriam Armant ; Colleen Dansereau ; Troy C. Lund ; Weston P. Miller ; Gerald V. Raymond ; Raman Sankar ; Ami J. Shah ; Caroline Sevin ; H Bobby Gaspar ; Paul Gissen ; Hernan Amartino ; Drago Bratkovic ; Nicholas J C. Smith ; Asif M. Paker ; Esther Shamir ; Tara O'Meara ; David Davidson ; Patrick Aubourg ; David A. Williams

Source :

The New England journal of medicine [ 1533-4406 ] ; 2017.

RBID : pubmed:28976817

Descripteurs français

English descriptors

KwdEn :
- ATP-Binding Cassette Transporters (genetics), ATP-Binding Cassette Transporters (therapeutic use), Adolescent, Adrenoleukodystrophy (genetics), Adrenoleukodystrophy (therapy), Antigens, CD34 (blood), Biomarkers (blood), Child, Combined Modality Therapy, Genetic Therapy, Genetic Vectors (blood), Granulocyte Colony-Stimulating Factor (therapeutic use), Hematopoietic Stem Cell Transplantation, Hematopoietic Stem Cells (immunology), Humans, Lentivirus, Male, Polymerase Chain Reaction, Transplantation, Autologous.
MESH :
- chemical , blood : Antigens, CD34, Biomarkers.
- blood : Genetic Vectors.
- genetics : ATP-Binding Cassette Transporters, Adrenoleukodystrophy.
- immunology : Hematopoietic Stem Cells.
- therapeutic use : ATP-Binding Cassette Transporters, Granulocyte Colony-Stimulating Factor.
- therapy : Adrenoleukodystrophy.
- Adolescent, Child, Combined Modality Therapy, Genetic Therapy, Hematopoietic Stem Cell Transplantation, Humans, Lentivirus, Male, Polymerase Chain Reaction, Transplantation, Autologous.

Abstract

In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allogeneic hematopoietic stem-cell transplantation.

DOI: 10.1056/NEJMoa1700554
PubMed: 28976817

Affiliations:

Le document en format XML

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<author><name sortKey="Eichler, Florian" sort="Eichler, Florian" uniqKey="Eichler F" first="Florian" last="Eichler">Florian Eichler</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
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<author><name sortKey="Duncan, Christine" sort="Duncan, Christine" uniqKey="Duncan C" first="Christine" last="Duncan">Christine Duncan</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
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<author><name sortKey="Musolino, Patricia L" sort="Musolino, Patricia L" uniqKey="Musolino P" first="Patricia L" last="Musolino">Patricia L. Musolino</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Orchard, Paul J" sort="Orchard, Paul J" uniqKey="Orchard P" first="Paul J" last="Orchard">Paul J. Orchard</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="De Oliveira, Satiro" sort="De Oliveira, Satiro" uniqKey="De Oliveira S" first="Satiro" last="De Oliveira">Satiro De Oliveira</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Thrasher, Adrian J" sort="Thrasher, Adrian J" uniqKey="Thrasher A" first="Adrian J" last="Thrasher">Adrian J. Thrasher</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Armant, Myriam" sort="Armant, Myriam" uniqKey="Armant M" first="Myriam" last="Armant">Myriam Armant</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Dansereau, Colleen" sort="Dansereau, Colleen" uniqKey="Dansereau C" first="Colleen" last="Dansereau">Colleen Dansereau</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Lund, Troy C" sort="Lund, Troy C" uniqKey="Lund T" first="Troy C" last="Lund">Troy C. Lund</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Miller, Weston P" sort="Miller, Weston P" uniqKey="Miller W" first="Weston P" last="Miller">Weston P. Miller</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Raymond, Gerald V" sort="Raymond, Gerald V" uniqKey="Raymond G" first="Gerald V" last="Raymond">Gerald V. Raymond</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Sankar, Raman" sort="Sankar, Raman" uniqKey="Sankar R" first="Raman" last="Sankar">Raman Sankar</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Shah, Ami J" sort="Shah, Ami J" uniqKey="Shah A" first="Ami J" last="Shah">Ami J. Shah</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Sevin, Caroline" sort="Sevin, Caroline" uniqKey="Sevin C" first="Caroline" last="Sevin">Caroline Sevin</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Gaspar, H Bobby" sort="Gaspar, H Bobby" uniqKey="Gaspar H" first="H Bobby" last="Gaspar">H Bobby Gaspar</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Gissen, Paul" sort="Gissen, Paul" uniqKey="Gissen P" first="Paul" last="Gissen">Paul Gissen</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Amartino, Hernan" sort="Amartino, Hernan" uniqKey="Amartino H" first="Hernan" last="Amartino">Hernan Amartino</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Bratkovic, Drago" sort="Bratkovic, Drago" uniqKey="Bratkovic D" first="Drago" last="Bratkovic">Drago Bratkovic</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Smith, Nicholas J C" sort="Smith, Nicholas J C" uniqKey="Smith N" first="Nicholas J C" last="Smith">Nicholas J C. Smith</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Paker, Asif M" sort="Paker, Asif M" uniqKey="Paker A" first="Asif M" last="Paker">Asif M. Paker</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Shamir, Esther" sort="Shamir, Esther" uniqKey="Shamir E" first="Esther" last="Shamir">Esther Shamir</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="O Meara, Tara" sort="O Meara, Tara" uniqKey="O Meara T" first="Tara" last="O'Meara">Tara O'Meara</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Davidson, David" sort="Davidson, David" uniqKey="Davidson D" first="David" last="Davidson">David Davidson</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Aubourg, Patrick" sort="Aubourg, Patrick" uniqKey="Aubourg P" first="Patrick" last="Aubourg">Patrick Aubourg</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Williams, David A" sort="Williams, David A" uniqKey="Williams D" first="David A" last="Williams">David A. Williams</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
</titleStmt>
<publicationStmt><idno type="wicri:source">PubMed</idno>
<date when="2017">2017</date>
<idno type="RBID">pubmed:28976817</idno>
<idno type="pmid">28976817</idno>
<idno type="doi">10.1056/NEJMoa1700554</idno>
<idno type="wicri:Area/PubMed/Corpus">000394</idno>
<idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">000394</idno>
<idno type="wicri:Area/PubMed/Curation">000392</idno>
<idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Curation">000392</idno>
<idno type="wicri:Area/PubMed/Checkpoint">000392</idno>
<idno type="wicri:explorRef" wicri:stream="Checkpoint" wicri:step="PubMed">000392</idno>
<idno type="wicri:Area/Ncbi/Merge">004F85</idno>
<idno type="wicri:Area/Ncbi/Curation">004F85</idno>
<idno type="wicri:Area/Ncbi/Checkpoint">004F85</idno>
<idno type="wicri:Area/Main/Merge">000A95</idno>
<idno type="wicri:Area/Main/Curation">000B00</idno>
<idno type="wicri:Area/Main/Exploration">000B00</idno>
</publicationStmt>
<sourceDesc><biblStruct><analytic><title xml:lang="en">Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.</title>
<author><name sortKey="Eichler, Florian" sort="Eichler, Florian" uniqKey="Eichler F" first="Florian" last="Eichler">Florian Eichler</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Duncan, Christine" sort="Duncan, Christine" uniqKey="Duncan C" first="Christine" last="Duncan">Christine Duncan</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Musolino, Patricia L" sort="Musolino, Patricia L" uniqKey="Musolino P" first="Patricia L" last="Musolino">Patricia L. Musolino</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Orchard, Paul J" sort="Orchard, Paul J" uniqKey="Orchard P" first="Paul J" last="Orchard">Paul J. Orchard</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="De Oliveira, Satiro" sort="De Oliveira, Satiro" uniqKey="De Oliveira S" first="Satiro" last="De Oliveira">Satiro De Oliveira</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Thrasher, Adrian J" sort="Thrasher, Adrian J" uniqKey="Thrasher A" first="Adrian J" last="Thrasher">Adrian J. Thrasher</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Armant, Myriam" sort="Armant, Myriam" uniqKey="Armant M" first="Myriam" last="Armant">Myriam Armant</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Dansereau, Colleen" sort="Dansereau, Colleen" uniqKey="Dansereau C" first="Colleen" last="Dansereau">Colleen Dansereau</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Lund, Troy C" sort="Lund, Troy C" uniqKey="Lund T" first="Troy C" last="Lund">Troy C. Lund</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Miller, Weston P" sort="Miller, Weston P" uniqKey="Miller W" first="Weston P" last="Miller">Weston P. Miller</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Raymond, Gerald V" sort="Raymond, Gerald V" uniqKey="Raymond G" first="Gerald V" last="Raymond">Gerald V. Raymond</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Sankar, Raman" sort="Sankar, Raman" uniqKey="Sankar R" first="Raman" last="Sankar">Raman Sankar</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Shah, Ami J" sort="Shah, Ami J" uniqKey="Shah A" first="Ami J" last="Shah">Ami J. Shah</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Sevin, Caroline" sort="Sevin, Caroline" uniqKey="Sevin C" first="Caroline" last="Sevin">Caroline Sevin</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Gaspar, H Bobby" sort="Gaspar, H Bobby" uniqKey="Gaspar H" first="H Bobby" last="Gaspar">H Bobby Gaspar</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Gissen, Paul" sort="Gissen, Paul" uniqKey="Gissen P" first="Paul" last="Gissen">Paul Gissen</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Amartino, Hernan" sort="Amartino, Hernan" uniqKey="Amartino H" first="Hernan" last="Amartino">Hernan Amartino</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Bratkovic, Drago" sort="Bratkovic, Drago" uniqKey="Bratkovic D" first="Drago" last="Bratkovic">Drago Bratkovic</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Smith, Nicholas J C" sort="Smith, Nicholas J C" uniqKey="Smith N" first="Nicholas J C" last="Smith">Nicholas J C. Smith</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Paker, Asif M" sort="Paker, Asif M" uniqKey="Paker A" first="Asif M" last="Paker">Asif M. Paker</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Shamir, Esther" sort="Shamir, Esther" uniqKey="Shamir E" first="Esther" last="Shamir">Esther Shamir</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="O Meara, Tara" sort="O Meara, Tara" uniqKey="O Meara T" first="Tara" last="O'Meara">Tara O'Meara</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Davidson, David" sort="Davidson, David" uniqKey="Davidson D" first="David" last="Davidson">David Davidson</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Aubourg, Patrick" sort="Aubourg, Patrick" uniqKey="Aubourg P" first="Patrick" last="Aubourg">Patrick Aubourg</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
<author><name sortKey="Williams, David A" sort="Williams, David A" uniqKey="Williams D" first="David A" last="Williams">David A. Williams</name>
<affiliation><nlm:affiliation>From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).</nlm:affiliation>
<wicri:noCountry code="subField">N.J.C.S.).</wicri:noCountry>
</affiliation>
</author>
</analytic>
<series><title level="j">The New England journal of medicine</title>
<idno type="eISSN">1533-4406</idno>
<imprint><date when="2017" type="published">2017</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
</fileDesc>
<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>ATP-Binding Cassette Transporters (genetics)</term>
<term>ATP-Binding Cassette Transporters (therapeutic use)</term>
<term>Adolescent</term>
<term>Adrenoleukodystrophy (genetics)</term>
<term>Adrenoleukodystrophy (therapy)</term>
<term>Antigens, CD34 (blood)</term>
<term>Biomarkers (blood)</term>
<term>Child</term>
<term>Combined Modality Therapy</term>
<term>Genetic Therapy</term>
<term>Genetic Vectors (blood)</term>
<term>Granulocyte Colony-Stimulating Factor (therapeutic use)</term>
<term>Hematopoietic Stem Cell Transplantation</term>
<term>Hematopoietic Stem Cells (immunology)</term>
<term>Humans</term>
<term>Lentivirus</term>
<term>Male</term>
<term>Polymerase Chain Reaction</term>
<term>Transplantation, Autologous</term>
</keywords>
<keywords scheme="KwdFr" xml:lang="fr"><term>Adolescent</term>
<term>Adrénoleucodystrophie ()</term>
<term>Adrénoleucodystrophie (génétique)</term>
<term>Antigènes CD34 (sang)</term>
<term>Association thérapeutique</term>
<term>Cellules souches hématopoïétiques (immunologie)</term>
<term>Enfant</term>
<term>Facteur de stimulation  des colonies de granulocytes (usage thérapeutique)</term>
<term>Humains</term>
<term>Lentivirus</term>
<term>Marqueurs biologiques (sang)</term>
<term>Mâle</term>
<term>Réaction de polymérisation en chaîne</term>
<term>Thérapie génétique</term>
<term>Transplantation autologue</term>
<term>Transplantation de cellules souches hématopoïétiques</term>
<term>Transporteurs ABC (génétique)</term>
<term>Transporteurs ABC (usage thérapeutique)</term>
<term>Vecteurs génétiques (sang)</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="blood" xml:lang="en"><term>Antigens, CD34</term>
<term>Biomarkers</term>
</keywords>
<keywords scheme="MESH" qualifier="blood" xml:lang="en"><term>Genetic Vectors</term>
</keywords>
<keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>ATP-Binding Cassette Transporters</term>
<term>Adrenoleukodystrophy</term>
</keywords>
<keywords scheme="MESH" qualifier="génétique" xml:lang="fr"><term>Adrénoleucodystrophie</term>
<term>Transporteurs ABC</term>
</keywords>
<keywords scheme="MESH" qualifier="immunologie" xml:lang="fr"><term>Cellules souches hématopoïétiques</term>
</keywords>
<keywords scheme="MESH" qualifier="immunology" xml:lang="en"><term>Hematopoietic Stem Cells</term>
</keywords>
<keywords scheme="MESH" qualifier="sang" xml:lang="fr"><term>Antigènes CD34</term>
<term>Marqueurs biologiques</term>
<term>Vecteurs génétiques</term>
</keywords>
<keywords scheme="MESH" qualifier="therapeutic use" xml:lang="en"><term>ATP-Binding Cassette Transporters</term>
<term>Granulocyte Colony-Stimulating Factor</term>
</keywords>
<keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Adrenoleukodystrophy</term>
</keywords>
<keywords scheme="MESH" qualifier="usage thérapeutique" xml:lang="fr"><term>Facteur de stimulation  des colonies de granulocytes</term>
<term>Transporteurs ABC</term>
</keywords>
<keywords scheme="MESH" xml:lang="en"><term>Adolescent</term>
<term>Child</term>
<term>Combined Modality Therapy</term>
<term>Genetic Therapy</term>
<term>Hematopoietic Stem Cell Transplantation</term>
<term>Humans</term>
<term>Lentivirus</term>
<term>Male</term>
<term>Polymerase Chain Reaction</term>
<term>Transplantation, Autologous</term>
</keywords>
<keywords scheme="MESH" xml:lang="fr"><term>Adolescent</term>
<term>Adrénoleucodystrophie</term>
<term>Association thérapeutique</term>
<term>Enfant</term>
<term>Humains</term>
<term>Lentivirus</term>
<term>Mâle</term>
<term>Réaction de polymérisation en chaîne</term>
<term>Thérapie génétique</term>
<term>Transplantation autologue</term>
<term>Transplantation de cellules souches hématopoïétiques</term>
</keywords>
</textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en">In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allogeneic hematopoietic stem-cell transplantation.</div>
</front>
</TEI>
<affiliations><list></list>
<tree><noCountry><name sortKey="Amartino, Hernan" sort="Amartino, Hernan" uniqKey="Amartino H" first="Hernan" last="Amartino">Hernan Amartino</name>
<name sortKey="Armant, Myriam" sort="Armant, Myriam" uniqKey="Armant M" first="Myriam" last="Armant">Myriam Armant</name>
<name sortKey="Aubourg, Patrick" sort="Aubourg, Patrick" uniqKey="Aubourg P" first="Patrick" last="Aubourg">Patrick Aubourg</name>
<name sortKey="Bratkovic, Drago" sort="Bratkovic, Drago" uniqKey="Bratkovic D" first="Drago" last="Bratkovic">Drago Bratkovic</name>
<name sortKey="Dansereau, Colleen" sort="Dansereau, Colleen" uniqKey="Dansereau C" first="Colleen" last="Dansereau">Colleen Dansereau</name>
<name sortKey="Davidson, David" sort="Davidson, David" uniqKey="Davidson D" first="David" last="Davidson">David Davidson</name>
<name sortKey="De Oliveira, Satiro" sort="De Oliveira, Satiro" uniqKey="De Oliveira S" first="Satiro" last="De Oliveira">Satiro De Oliveira</name>
<name sortKey="Duncan, Christine" sort="Duncan, Christine" uniqKey="Duncan C" first="Christine" last="Duncan">Christine Duncan</name>
<name sortKey="Eichler, Florian" sort="Eichler, Florian" uniqKey="Eichler F" first="Florian" last="Eichler">Florian Eichler</name>
<name sortKey="Gaspar, H Bobby" sort="Gaspar, H Bobby" uniqKey="Gaspar H" first="H Bobby" last="Gaspar">H Bobby Gaspar</name>
<name sortKey="Gissen, Paul" sort="Gissen, Paul" uniqKey="Gissen P" first="Paul" last="Gissen">Paul Gissen</name>
<name sortKey="Lund, Troy C" sort="Lund, Troy C" uniqKey="Lund T" first="Troy C" last="Lund">Troy C. Lund</name>
<name sortKey="Miller, Weston P" sort="Miller, Weston P" uniqKey="Miller W" first="Weston P" last="Miller">Weston P. Miller</name>
<name sortKey="Musolino, Patricia L" sort="Musolino, Patricia L" uniqKey="Musolino P" first="Patricia L" last="Musolino">Patricia L. Musolino</name>
<name sortKey="O Meara, Tara" sort="O Meara, Tara" uniqKey="O Meara T" first="Tara" last="O'Meara">Tara O'Meara</name>
<name sortKey="Orchard, Paul J" sort="Orchard, Paul J" uniqKey="Orchard P" first="Paul J" last="Orchard">Paul J. Orchard</name>
<name sortKey="Paker, Asif M" sort="Paker, Asif M" uniqKey="Paker A" first="Asif M" last="Paker">Asif M. Paker</name>
<name sortKey="Raymond, Gerald V" sort="Raymond, Gerald V" uniqKey="Raymond G" first="Gerald V" last="Raymond">Gerald V. Raymond</name>
<name sortKey="Sankar, Raman" sort="Sankar, Raman" uniqKey="Sankar R" first="Raman" last="Sankar">Raman Sankar</name>
<name sortKey="Sevin, Caroline" sort="Sevin, Caroline" uniqKey="Sevin C" first="Caroline" last="Sevin">Caroline Sevin</name>
<name sortKey="Shah, Ami J" sort="Shah, Ami J" uniqKey="Shah A" first="Ami J" last="Shah">Ami J. Shah</name>
<name sortKey="Shamir, Esther" sort="Shamir, Esther" uniqKey="Shamir E" first="Esther" last="Shamir">Esther Shamir</name>
<name sortKey="Smith, Nicholas J C" sort="Smith, Nicholas J C" uniqKey="Smith N" first="Nicholas J C" last="Smith">Nicholas J C. Smith</name>
<name sortKey="Thrasher, Adrian J" sort="Thrasher, Adrian J" uniqKey="Thrasher A" first="Adrian J" last="Thrasher">Adrian J. Thrasher</name>
<name sortKey="Williams, David A" sort="Williams, David A" uniqKey="Williams D" first="David A" last="Williams">David A. Williams</name>
</noCountry>
</tree>
</affiliations>
</record>

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   |area=    AustralieFrV1
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   |texte=   Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
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Serveur d'exploration sur les relations entre la France et l'Australie

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

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	Serveur d'exploration sur les relations entre la France et l'Australie
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