Gene therapy in patients with transfusion-dependent β-thalassemia

New England Journal of Medicine

Volumn 378, Issue 16, 2018, Pages 1479-1493

  Thompson, A A ^a   Walters, M C ^b   Kwiatkowski, J ^e   Rasko, J E J ^f,g,h   Ribeil, J A ^i,j,s   Hongeng, S ^u   Magrin, E ^i,k   Schiller, G J ^d   Payen, E ^o   Semeraro, M ^k,l   Moshous, D ^i,k   Lefrere, F ⁱ   Puy, H ^l,p   Bourget, P ⁱ   Magnani, A ^i,j   Caccavelli, L ^i,j   Diana, J S ⁱ   Suarez, F ⁱ   Monpoux, F ⁱ   Brousse, V ⁱ   Poirot, C ^l,m   Brouzes, C ⁱ   Meritet, J F ⁿ   Pondarre C ^q   Beuzard, Y ^o   Chretien S ^o   Lefebvre, T ^l,p   Teachey, D T ^e   Anurathapan, U ^u   Ho, P J ^g,h   Von Kalle, C ^v   Kletzel, M ^a   Vichinsky, E ^b   Soni, S ^c,s   Veres, G ^s   Negre, O ^s   Ross, R W ^s   Davidson, D ^s   Petrusich, A ^s   Sandler, L ^s   Asmal, M ^s   Hermine, O ⁱ   De Montalembert, M ⁱ   Hacein Bey Abina, S ^l,r   Blanche, S ⁱ   Leboulch, P ^o,t,u   Cavazzana, M ^i,j,k   more..

^a NORTHWESTERN UNIVERSITY   (United States)

^b CHILDREN S HOSPITAL OAKLAND   (United States)

^c LUCILE PACKARD CHILDREN S HOSPITAL   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e UNIVERSITY OF PENNSYLVANIA   (United States)

^f CENTENARY INSTITUTE   (Australia)

^g UNIVERSITY OF SYDNEY   (Australia)

^h ROYAL PRINCE ALFRED HOSPITAL   (Australia)

ⁱ HÔPITAL NECKER ENFANTS MALADES   (France)

^j Clinical Investigation Center in Biotherapy   (France)

^k Imagine Institute   (France)

^l UNIVERSITÉ PARIS DESCARTES   (France)

^m SORBONNE UNIVERSITÉ   (France)

ⁿ Pierre et Marie Curie University ^*  (France)

^o UNIVERSITÉ PARIS SACLAY   (France)

^p HÔPITAL LOUIS MOURIER   (France)

^q CENTRE HOSPITALIER INTERCOMMUNAL DE CRÉTEIL   (France)

^r BICÊTRE HOSPITAL   (France)

^s BLUEBIRD BIO   (United States)

^t BRIGHAM AND WOMEN S HOSPITAL   (United States)

^u MAHIDOL UNIVERSITY   (Thailand)

^v GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

more..

Author keywords

[No Author keywords available]

Indexed keywords

BUSULFAN; HEMOGLOBIN; HEMOGLOBIN A; LENTIGLOBIN BB305; CD34 ANTIGEN; HEMOGLOBIN BETA CHAIN;

ADOLESCENT; ADULT; ARTICLE; AUTOLOGOUS STEM CELL TRANSPLANTATION; BETA THALASSEMIA; CHILD; CLINICAL ARTICLE; CLINICAL EFFECTIVENESS; CONTROLLED STUDY; COPY NUMBER VARIATION; DYSERYTHROPOIESIS; ERYTHROCYTE TRANSFUSION; FEMALE; GENE THERAPY; GENOTYPE; HEMOGLOBIN BLOOD LEVEL; HUMAN; HUMAN CELL; LIVER VENOOCCLUSIVE DISEASE; MALE; MYELOABLATIVE CONDITIONING; OPEN STUDY; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; SAFETY; YOUNG ADULT; AUTOTRANSPLANTATION; CLINICAL TRIAL; GENE TRANSFER; GENE VECTOR; GENETICS; LENTIVIRUS; MUTATION; UTILIZATION;

ADOLESCENT; ADULT; ANTIGENS, CD34; BETA-GLOBINS; BETA-THALASSEMIA; CHILD; ERYTHROCYTE TRANSFUSION; FEMALE; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HEMOGLOBINS; HUMANS; LENTIVIRUS; MALE; MUTATION; TRANSPLANTATION, AUTOLOGOUS; YOUNG ADULT;

EID: 85045833861     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1705342     Document Type: Article

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