Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Science Translational Medicine

Volumn 8, Issue 335, 2016, Pages

  De Ravin, Suk See ^a   Wu, Xiaolin ^b   Moir, Susan ^a   Anaya O'Brien, Sandra ^a   Kwatemaa, Nana ^a   Littel, Patricia ^a   Theobald, Narda ^a   Choi, Uimook ^a   Su, Ling ^b   Marquesen, Martha ^a   Hilligoss, Dianne ^a   Lee, Janet ^a   Buckner, Clarissa M ^a   Zarember, Kol A ^a   O'Connor, Geraldine ^c   McVicar, Daniel ^c   Kuhns, Douglas ^b   Throm, Robert E ^d   Zhou, Sheng ^d   Notarangelo, Luigi D ^e   Hanson, I Celine ^f   Cowan, Mort J ^g   Kang, Elizabeth ^a   Hadigan, Coleen ^a   Meagher, Michael ^d   Gray, John T ^h   Sorrentino, Brian P ^d   Malech, Harry L ^a   more..

^a NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^b FREDERICK NATIONAL LABORATORY FOR CANCER RESEARCH   (United States)

^c NATIONAL CANCER INSTITUTE   (United States)

^d ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^e BOSTON CHILDREN S HOSPITAL   (United States)

^f TEXAS CHILDREN S HOSPITAL   (United States)

^g UNIVERSITY OF CALIFORNIA   (United States)

^h Audentes Therapeutics   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BUSULFAN; ELONGATION FACTOR 1ALPHA; LENTIVIRUS VECTOR; IL2RG PROTEIN, HUMAN; INTERLEUKIN 2 RECEPTOR GAMMA;

ADOLESCENT; ADULT; ARTICLE; B LYMPHOCYTE; CELL BASED GENE THERAPY; CELL EXPANSION; CHILD; CLINICAL ARTICLE; CLONAL VARIATION; CONTROLLED STUDY; FOLLOW UP; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; HUMORAL IMMUNITY; IMMUNIZATION; IMMUNOSUPPRESSIVE TREATMENT; MALE; NATURAL KILLER CELL; PRIORITY JOURNAL; SCHOOL CHILD; T LYMPHOCYTE; VIRAL GENE THERAPY; X LINKED SEVERE COMBINED IMMUNODEFICIENCY; YOUNG ADULT; CLINICAL TRIAL; GENE THERAPY; GENE VECTOR; GENETICS; LENTIVIRINAE; METABOLISM; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PROCEDURES;

ADOLESCENT; ADULT; B-LYMPHOCYTES; CHILD; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HUMANS; INTERLEUKIN RECEPTOR COMMON GAMMA SUBUNIT; KILLER CELLS, NATURAL; LENTIVIRUS; MALE; T-LYMPHOCYTES; X-LINKED COMBINED IMMUNODEFICIENCY DISEASES; YOUNG ADULT;

EID: 84964514054     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.aad8856     Document Type: Article

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