Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy

New England Journal of Medicine

Volumn 377, Issue 17, 2017, Pages 1630-1638

  Eichler, Florian ^a   Duncan, Christine ^b   Musolino, Patricia L ^a   Orchard, Paul J ^e   De Oliveira, Satiro ^f   Thrasher, Adrian J ^g   Armant, Myriam ^b   Dansereau, Colleen ^b   Lund, Troy C ^e   Miller, Weston P ^e   Raymond, Gerald V ^e   Sankar, Raman ^f   Shah, Ami J ^f   Sevin, Caroline ^h   Gaspar, H Bobby ^g   Gissen, Paul ^g   Amartino, Hernan ⁱ   Bratkovic, Drago ^j   Smith, Nicholas J C ^j   Paker, Asif M ^d   Shamir, Esther ^d   O’Meara, Tara ^d   Davidson, David ^d   Aubourg, Patrick ^h   Williams, David A ^b,c   more..

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b DANA FARBER CANCER INSTITUTE   (United States)

^c BOSTON CHILDREN S HOSPITAL   (United States)

^d BLUEBIRD BIO   (United States)

^e UNIVERSITY OF MINNESOTA   (United States)

^f UNIVERSITY OF CALIFORNIA   (United States)

^g GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^h BICÊTRE HOSPITAL   (France)

ⁱ Foundation investigar   (Argentina)

^j WOMEN S AND CHILDREN S HOSPITAL   (Australia)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ABC TRANSPORTER; ALD PROTEIN; BUSULFAN; CD34 ANTIGEN; CYCLOPHOSPHAMIDE; ELIVALDOGENE TAVALENTIVEC; GRANULOCYTE COLONY STIMULATING FACTOR; UNCLASSIFIED DRUG; ABCD1 PROTEIN, HUMAN; BIOLOGICAL MARKER;

ADOLESCENT; ADRENOLEUKODYSTROPHY; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BRAIN DISEASE; CEREBRAL ADRENOLEUKODYSTROPHY; CHILD; CLINICAL ARTICLE; CLINICAL OUTCOME; DRUG EFFICACY; DRUG SAFETY; ENGRAFTMENT; EXPERIMENTAL THERAPY; FOLLOW UP; GENE THERAPY; HUMAN; MALE; MULTICENTER STUDY; NUCLEAR MAGNETIC RESONANCE IMAGING; PHASE 2 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; AUTOTRANSPLANTATION; BLOOD; CLINICAL TRIAL; GENE VECTOR; GENETICS; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IMMUNOLOGY; LENTIVIRINAE; MULTIMODALITY CANCER THERAPY; POLYMERASE CHAIN REACTION;

ADOLESCENT; ADRENOLEUKODYSTROPHY; ANTIGENS, CD34; ATP-BINDING CASSETTE TRANSPORTERS; BIOMARKERS; CHILD; COMBINED MODALITY THERAPY; GENETIC THERAPY; GENETIC VECTORS; GRANULOCYTE COLONY-STIMULATING FACTOR; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; LENTIVIRUS; MALE; POLYMERASE CHAIN REACTION; TRANSPLANTATION, AUTOLOGOUS;

EID: 85032463285     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1700554     Document Type: Article

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