Evolving Gene Therapy in Primary Immunodeficiency

Molecular Therapy

Volumn 25, Issue 5, 2017, Pages 1132-1141

  Thrasher, Adrian J ^a,b   Williams, David A ^c  

^a GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c BOSTON CHILDREN S HOSPITAL   (United States)

Author keywords

gene therapy; immunodeficiencies; retrovirus

Indexed keywords

ADENOSINE DEAMINASE; BUSULFAN; CD40 LIGAND; IMMUNOGLOBULIN; LENTIVIRUS VECTOR; MACROGOL; MELPHALAN; RETROVIRUS VECTOR; STAT1 PROTEIN; STAT3 PROTEIN;

ACUTE LYMPHOBLASTIC LEUKEMIA; ADENOSINE DEAMINASE DEFICIENCY; AUTOLOGOUS STEM CELL TRANSPLANTATION; BCL2 ONCOGENE; BTK GENE; CARCINOGENESIS; CCND2 ONCOGENE; CELL REPROGRAMMING TECHNIQUE; CHRONIC GRANULOMATOUS DISEASE; CLINICAL EFFECTIVENESS; CPG ISLAND; CYBB GENE; DCLRE1C GENE; DISEASE FREE SURVIVAL; DNA METHYLATION; DRUG SAFETY; GENE EDITING; GENE LOCUS; GENE THERAPY; GENE TRANSFER; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HSC GENE; HUMAN; IL2R GENE; IMMUNE DEFICIENCY; IMMUNOPATHOGENESIS; ITGB2 GENE; JAK3 DEFICIENCY; JAK3 GENE; LEUCOCYTE ADHESION DEFICIENCY TYPE 1; LEUKEMOGENESIS; LMO2 ONCOGENE; LONG TERMINAL REPEAT; LYMPHOCYTE; MECOM GENE; MEMBRANE RECONSTITUTION; MORBIDITY; MORTALITY; MUTAGENESIS; NCF1 PSEUDOGENE; NONHUMAN; ONCOGENE; OVERALL SURVIVAL; PRDM16 GENE; RAG GENE; REDUCED INTENSITY CONDITIONING; REVIEW; SEVERE COMBINED IMMUNODEFICIENCY; SURVIVAL RATE; TNFSF5 GENE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; WAS GENE; WISKOTT ALDRICH SYNDROME; X LINKED AGAMMAGLOBULINEMIA; X LINKED HYPER IMMUNOGLOBULIN M SYNDROME; ALLOTRANSPLANTATION; CHEMISTRY; CLINICAL TRIAL (TOPIC); DEFICIENCY; GAMMARETROVIRUS; GENE EXPRESSION; GENE VECTOR; GENETICS; HISTORY; IMMUNOLOGY; LENTIVIRUS; PATHOLOGY; PROCEDURES;

ADENOSINE DEAMINASE; CLINICAL TRIALS AS TOPIC; GAMMARETROVIRUS; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; LENTIVIRUS; SEVERE COMBINED IMMUNODEFICIENCY; TRANSPLANTATION, HOMOLOGOUS;

EID: 85018781253     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.03.018     Document Type: Review

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