Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome

JAMA - Journal of the American Medical Association

Volumn 313, Issue 15, 2015, Pages 1550-1563

  Hacein Bey Abina, Salima ^a,b,c,d   Gaspar, H Bobby ^e,f   Blondeau, Johanna ^a,b   Caccavelli, Laure ^a,b   Charrier, Sabine ^g,l   Buckland, Karen ^e,f   Picard, Capucine ^a,b,h   Six, Emmanuelle ^h,i   Himoudi, Nourredine ^e,f   Gilmour, Kimberly ^e,f   McNicol, Anne Marie ^e,f   Hara, Havinder ^e,f   Xu Bayford, Jinhua ^f   Rivat, Christine ^e,f   Touzot, Fabien ^a,b,h   Mavilio, Fulvio ^g,l   Lim, Annick ^j   Treluyer, Jean Marc ^a   Héritier, Sébastien ^b,h   Lefrère, Francois ^a   Magalon, Jeremy ^a,b   Pengue Koyi, Isabelle ^a,b,h   Honnet, Géraldine ^g,l   Blanche, Stéphane ^b,h   Sherman, Eric A ^a,m   Male, Frances ^a,m   Berry, Charles ^a,m   Malani, Nirav ^a,m   Bushman, Frederic D ^a,m   Fischer, Alain ^a,b,h,i,k   Thrasher, Adrian J ^e,f   Galy, Anne ^g,l   Cavazzana, Marina ^a,b,h   more..

^a HÔPITAL NECKER ENFANTS MALADES   (France)

^b ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

^c Centre National de la Recherche Scientifique   (France)

^d UNIVERSITÉ PARIS SACLAY   (France)

^e UNIVERSITY COLLEGE LONDON   (United Kingdom)

^f National Health Service Foundation Trust   (United Kingdom)

^g UNIVERSITÉ D EVRY VAL D ESSONNE   (France)

^h INSERM US24 CNRS UMS3633   (France)

ⁱ INSERM   (France)

^j INSTITUT PASTEUR   (France)

^k Paris Sciences et Lettres   (France)

^l GÉNÉTHON   (France)

^m UNIVERSITY OF PENNSYLVANIA   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

HLA ANTIGEN; LENTIVIRUS VECTOR; WISKOTT ALDRICH SYNDROME PROTEIN;

ADOLESCENT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTHRITIS; ARTICLE; AUTOIMMUNE DISEASE; BACTERIAL INFECTION; BLEEDING; BRAIN HEMORRHAGE; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; CYTOPENIA; DISEASE SEVERITY; ECZEMA; GASTROINTESTINAL HEMORRHAGE; GENERALIZED LYMPHADENOPATHY; HEPATOMEGALY; HERPES VIRUS INFECTION; HOSPITALIZATION; HUMAN; HUMAN CELL; INFECTION; INFLAMMATION; LYMPHOMA; LYMPHOPROLIFERATIVE DISEASE; MORBIDITY; PRESCHOOL CHILD; PRIORITY JOURNAL; SCHOOL CHILD; SKIN INFECTION; STEM CELL GENE THERAPY; THROMBOCYTOPENIA; TREATMENT OUTCOME; VASCULITIS; VIRUS INFECTION; WISKOTT ALDRICH SYNDROME; ADVERSE EFFECTS; CLINICAL TRIAL; FEASIBILITY STUDY; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IMMUNOLOGY; INFANT; LENTIVIRINAE; MALE; MULTICENTER STUDY; NEWBORN; SEVERITY OF ILLNESS INDEX; WISKOTT-ALDRICH SYNDROME;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; FEASIBILITY STUDIES; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; INFANT, NEWBORN; LENTIVIRUS; MALE; SEVERITY OF ILLNESS INDEX; WISKOTT-ALDRICH SYNDROME; WISKOTT-ALDRICH SYNDROME PROTEIN FAMILY;

EID: 84928389399     PISSN: 00987484     EISSN: 15383598     Source Type: Journal    
DOI: 10.1001/jama.2015.3253     Document Type: Article

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