An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease

Molecular Therapy

Volumn 23, Issue 1, 2015, Pages 147-157

  Merling, Randall K ^a   Sweeney, Colin L ^a   Chu, Jessica ^a   Bodansky, Aaron ^a   Choi, Uimook ^a   Priel, Debra Long ^b   Kuhns, Douglas B ^b   Wang, Hongmei ^a   Vasilevsky, Sam ^a   De Ravin, Suk See ^a   Winkler, Thomas ^c   Dunbar, Cynthia E ^c   Zou, Jizhong ^d   Zarember, Kol A ^a   Gallin, John I ^a   Holland, Steven M ^a   Malech, Harry L ^a  

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

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

^c NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^d NATIONAL INSTITUTE OF ARTHRITIS AND MUSCULOSKELETAL AND SKIN DISEASES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; OXIDOREDUCTASE; REACTIVE OXYGEN METABOLITE; ZINC FINGER NUCLEASE; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE; ZINC FINGER PROTEIN;

ARTICLE; CELL DIFFERENTIATION; CHRONIC GRANULOMATOUS DISEASE; CONTROLLED STUDY; CYBA GENE; CYBB GENE; ENZYME ACTIVITY; GENE; GENE MUTATION; GENE TRANSFER; GENOTYPE; GRAM NEGATIVE BACTERIUM; GRANULIBACTER BETHESDENSIS; HEMATOPOIETIC STEM CELL; HOMOLOGOUS RECOMBINATION; HUMAN; MACROPHAGE; METABOLIC DISORDER; MONOCYTE; NCF1 GENE; NCF2 GENE; NCF4 GENE; NEUTROPHIL; NONHUMAN; PHAGOCYTE; PHAGOCYTOSIS; PLASMID; PLURIPOTENT STEM CELL; STAPHYLOCOCCUS AUREUS; ACETOBACTERACEAE; DEPENDOPARVOVIRUS; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETICS; GRANULOMATOUS DISEASE, CHRONIC; GROWTH, DEVELOPMENT AND AGING; IMMUNOLOGY; INDUCED PLURIPOTENT STEM CELL; METABOLISM; MICROBIOLOGY; PATHOLOGY; PROCEDURES;

BACTERIA (MICROORGANISMS); STAPHYLOCOCCUS AUREUS;

ACETOBACTERACEAE; CELL DIFFERENTIATION; DEPENDOVIRUS; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; GENOTYPE; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELLS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MACROPHAGES; NADPH OXIDASE; NEUTROPHILS; STAPHYLOCOCCUS AUREUS; ZINC FINGERS;

EID: 84920698348     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.195     Document Type: Article

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