Concise review: Lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases

Stem Cells Translational Medicine

Volumn 3, Issue 5, 2014, Pages 636-642

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

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Clinical trials; Gene therapy; Hematopoietic cells; Immunodeficiency; Progenitor cells

Indexed keywords

BUSULFAN; CD34 ANTIGEN; GRANULOCYTE COLONY STIMULATING FACTOR; INTERLEUKIN 15 RECEPTOR; INTERLEUKIN 2 RECEPTOR; INTERLEUKIN 21 RECEPTOR; INTERLEUKIN 4 RECEPTOR; INTERLEUKIN 7 RECEPTOR; INTERLEUKIN 9 RECEPTOR; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE;

ACUTE LYMPHOBLASTIC LEUKEMIA; ADENOSINE DEAMINASE DEFICIENCY; ALLOGENIC BONE MARROW TRANSPLANTATION; ARTICLE; CHRONIC GRANULOMATOUS DISEASE; CLINICAL TRIAL (TOPIC); ENZYME REPLACEMENT; GENE EXPRESSION; GENE THERAPY; GRAFT VERSUS HOST REACTION; HUMAN; HUMORAL IMMUNITY; IMMUNE DEFICIENCY; LEUKEMOGENESIS; MONOGENIC DISORDER; MUTAGENESIS; MYELODYSPLASTIC SYNDROME; NONHUMAN; RETROVIRUS; SEVERE COMBINED IMMUNODEFICIENCY; TUMOR SUPPRESSOR GENE; WISKOTT ALDRICH SYNDROME;

CLINICAL TRIALS; GENE THERAPY; HEMATOPOIETIC CELLS; IMMUNODEFICIENCY; PROGENITOR CELLS;

ANIMALS; COMMON VARIABLE IMMUNODEFICIENCY; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; ONCOGENE PROTEINS; TRANSCRIPTIONAL ACTIVATION;

EID: 84899675856     PISSN: 21576564     EISSN: 21576580     Source Type: Journal    
DOI: 10.5966/sctm.2013-0206     Document Type: Article

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