Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy

Brain

Volumn 141, Issue 1, 2018, Pages 99-116

  Holley, Rebecca J ^a   Ellison, Stuart M ^a   Fil, Daniel ^a   O'Leary, Claire ^a   McDermott, John ^a   Senthivel, Nishanthi ^a   Langford Smith, Alexander W W ^a,b   Wilkinson, Fiona L ^a,b   D'Souza, Zelpha ^a   Parker, Helen ^a   Liao, Aiyin ^a   Rowlston, Samuel ^a   Gleitz, Hélène F E ^a   Kan, Shih Hsin ^c   Dickson, Patricia I ^c   Bigger, Brian W ^a  

^a UNIVERSITY OF MANCHESTER   (United Kingdom)

^b MANCHESTER METROPOLITAN UNIVERSITY   (United Kingdom)

^c UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

lysosomal storage disease; microglia; mucopolysaccharidosis; neurodegeneration; stem cells

Indexed keywords

ALPHA N ACETYLGLUCOSAMINIDASE; CYTOKINE; ENZYME; HEPARAN SULFATE; INTERLEUKIN 1ALPHA; LENTIVIRUS VECTOR; MACROPHAGE INFLAMMATORY PROTEIN 1ALPHA; MONOCYTE CHEMOTACTIC PROTEIN 1; PREDNISOLONE; RANTES; GLYCOSAMINOGLYCAN; HEPARAN SULFATE SULFATASE; SULFATASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTOSIS; BONE MARROW TRANSPLANTATION; CONTROLLED STUDY; DISEASE SEVERITY; ENCEPHALITIS; ENZYME ACTIVITY; ENZYME RELEASE; EX VIVO STUDY; GLIOSIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; INFANT; LYSOSOME STORAGE DISEASE; MACROPHAGE; MOUSE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROBLEM BEHAVIOR; PROTEIN EXPRESSION; SANFILIPPO SYNDROME; STEM CELL GENE THERAPY; ANIMAL; BRAIN; C57BL MOUSE; COMPLICATION; DISEASE MODEL; ENZYMOLOGY; FEMALE; GENE THERAPY; GENETICS; HUMAN; LIVER; MALE; METABOLISM; PATHOLOGY; PHYSIOLOGY; PROCEDURES; SPLEEN; STEM CELL; TRANSGENIC MOUSE;

ANIMALS; BRAIN; CYTOKINES; DISEASE MODELS, ANIMAL; ENCEPHALITIS; FEMALE; GENETIC THERAPY; GLIOSIS; GLYCOSAMINOGLYCANS; HUMANS; LIVER; MACROPHAGES; MALE; MICE; MICE, INBRED C57BL; MICE, TRANSGENIC; MUCOPOLYSACCHARIDOSIS III; PREDNISOLONE; SPLEEN; STEM CELLS; SULFATASES;

EID: 85040644000     PISSN: 00068950     EISSN: 14602156     Source Type: Journal    
DOI: 10.1093/brain/awx311     Document Type: Article

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