Gene therapy for PIDs: Progress, pitfalls and prospects

Gene

Volumn 525, Issue 2, 2013, Pages 174-181

  Mukherjee, Sayandip ^a   Thrasher, Adrian J ^a,b  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b GREAT ORMOND STREET HOSPITAL   (United Kingdom)

Author keywords

CGD; Gene therapy; HSCT; PID; SCID; WAS

Indexed keywords

ADENOSINE DEAMINASE; BUSULFAN; FLUDARABINE; LENTIVIRUS VECTOR; MACROGOL; MELPHALAN; REACTIVE OXYGEN METABOLITE; RECOMBINANT INTERFERON; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE; RETROVIRUS VECTOR; RITUXIMAB; THYMOCYTE ANTIBODY; VIRUS VECTOR;

5' UNTRANSLATED REGION; ADENOSINE DEAMINASE DEFICIENCY; ALLOTRANSPLANTATION; AUTOIMMUNITY; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; B LYMPHOCYTE; CHRONIC GRANULOMATOUS DISEASE; CLINICAL TRIAL (TOPIC); DISEASE ASSOCIATION; DRUG EFFICACY; DRUG SAFETY; ENZYME REPLACEMENT; EX VIVO STUDY; FOLLOW UP; GENE TARGETING; GENOTOXICITY; HLA SYSTEM; HOMOLOGOUS RECOMBINATION; HUMAN; IMMUNE DEFICIENCY; MYELOABLATIVE CONDITIONING; NATURAL KILLER CELL; OUTCOME ASSESSMENT; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN TARGETING; RETROVIRUS; REVIEW; RISK BENEFIT ANALYSIS; SEVERE COMBINED IMMUNODEFICIENCY; SPLEEN FOCUS FORMING VIRUS; T LYMPHOCYTE; TISSUE SPECIFICITY; UMBILICAL CORD BLOOD; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; WISKOTT ALDRICH SYNDROME;

ADA-SCID; ADENOSINE DEAMINASE DEFICIENCY-SEVERE COMBINED IMMUNODEFICIENCY; CGD; CHRONIC GRANULOMATOUS DISORDER; DOUBLE STRAND BREAK; DSB; EF-1Α; ELONGATION FACTOR 1Α; ENZYME REPLACEMENT THERAPY; ERT; GALV; GENE THERAPY; GENOMIC SAFE HARBOUR; GIBBON APE LEUKAEMIA VIRUS; GRAFT VERSUS HOST DISEASE; GSH; GVHD; HAEMATOPOIETIC STEM CELL TRANSPLANT; HAEMATOPOIETIC STEM CELLS; HLA; HOMOLOGOUS RECOMBINATION; HR; HSCS; HSCT; HUMAN LEUKOCYTE ANTIGEN; IFN; IL; INTERFERON; INTERLEUKIN; LCR; LENTIVIRAL VECTORS; LOCUS CONTROL REGION; LONG TERMINAL REPEAT; LTR; LVS; MDS; MEGANUCLEASES; MICRORNA; MIR; MLV; MN; MURINE LEUKAEMIA VIRUS; MYELODYSPLASTIC SYNDROME; NADPH; NATURAL KILLER CELLS; NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE HYDROGEN; NK CELLS; PEG; PGK; PHOSPHOGLYCEROKINASE; PID; PIDS; POLYETHYLENE GLYCOL; PRIMARY IMMUNODEFICIENCY DISORDERS; REACTIVE OXYGEN SPECIES; RETROVIRAL VECTORS; ROS; RVS; SAE; SCID; SCID-X1; SELF-INACTIVATING; SERIOUS ADVERSE EVENT; SFFV; SIN; SPLEEN FOCUS-FORMING VIRUS; T CELL-ACUTE LYMPHOBLASTIC LEUKAEMIA; T-ALL; TALEN; TRANSCRIPTION ACTIVATOR-LIKE EFFECTOR NUCLEASES; UBIQUITOUS CHROMATIN OPENING ELEMENT; UCOE; VESICULAR STOMATITIS VIRUS-G PROTEIN; VSV-G; WAS; WASP; WISKOTT-ALDRICH SYNDROME; WISKOTT-ALDRICH SYNDROME PROTEIN; X-LINKED NEUTROPENIA; X-LINKED SEVERE COMBINED IMMUNODEFICIENCY; X-LINKED THROMBOCYTOPENIA; XLN; XLT; ZFN; ZINC FINGER NUCLEASE;

ADENOSINE DEAMINASE; AGAMMAGLOBULINEMIA; CLINICAL TRIALS AS TOPIC; GENETIC THERAPY; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; MUTAGENESIS, INSERTIONAL; SEVERE COMBINED IMMUNODEFICIENCY; TRANSGENES; TRANSPLANTATION, AUTOLOGOUS; X-LINKED COMBINED IMMUNODEFICIENCY DISEASES;

EID: 84879883823     PISSN: 03781119     EISSN: 18790038     Source Type: Journal    
DOI: 10.1016/j.gene.2013.03.098     Document Type: Review

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