Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning

Molecular Therapy

Volumn 19, Issue 10, 2011, Pages 1867-1877

  Huston, Marshall W ^a   Van Til, Niek P ^a   Visser, Trudi P ^a   Arshad, Shazia ^a   Brugman, Martijn H ^a,b   Cattoglio, Claudia ^c   Nowrouzi, Ali ^d   Li, Yuedan ^a   Schambach, Axel ^b   Schmidt, Manfred ^d   Baum, Christopher ^b   Von Kalle, Christof ^d   Mavilio, Fulvio ^c,e   Zhang, Fang ^f   Blundell, Mike P ^f   Thrasher, Adrian J ^f,g   Verstegen, Monique M A ^a   Wagemaker, Gerard ^a  

^a ERASMUS MEDICAL CENTER   (Netherlands)

^b HANNOVER MEDICAL SCHOOL   (Germany)

^c SAN RAFFAELE HOSPITAL   (Italy)

^d GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

^e UNIVERSITY OF MODENA AND REGGIO EMILIA   (Italy)

^f UNIVERSITY COLLEGE LONDON   (United Kingdom)

^g GREAT ORMOND STREET HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; CONCANAVALIN A; INTERLEUKIN 2; INTERLEUKIN 2 RECEPTOR; LENTIVIRUS VECTOR; PHOSPHOGLYCERATE KINASE; SELF INACTIVATING LENTIVIRUS VECTOR; T LYMPHOCYTE RECEPTOR; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY TITER; ARTICLE; B LYMPHOCYTE; CO GAMMA C GENE; CONTROLLED STUDY; GAMMA CPR GENE; GENE; GENE CONSTRUCT; GENE EXPRESSION REGULATION; GENE REARRANGEMENT; GENOTOXICITY; HUMAN; HUMAN CELL; HUMORAL IMMUNITY; IL2RG GENE; IMMUNOSUPPRESSIVE TREATMENT; LYMPHOCYTE ACTIVATION; LYMPHOCYTE PROLIFERATION; MALE; MEMBRANE RECONSTITUTION; MOUSE; NONHUMAN; OUTCOME ASSESSMENT; PROMOTER REGION; PROTO ONCOGENE; RISK REDUCTION; SPLEEN FOCUS FORMING VIRUS; T LYMPHOCYTE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

MURINAE; MUS; SPLEEN FOCUS-FORMING VIRUS;

EID: 80053568178     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.127     Document Type: Article

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