Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease

Molecular Therapy

Volumn 21, Issue 10, 2013, Pages 1938-1949

  Sergijenko, Ana ^a   Langford Smith, Alexander ^a   Liao, Ai Y ^a   Pickford, Claire E ^a   McDermott, John ^a   Nowinski, Gabriel ^a   Langford Smith, Kia J ^a   Merry, Catherine Lr ^a   Jones, Simon A ^b   Wraith, J Edmond ^b   Wynn, Robert F ^c   Wilkinson, Fiona L ^a   Bigger, Brian W ^a  

^a UNIVERSITY OF MANCHESTER   (United Kingdom)

^b ST MARY S HOSPITAL   (United Kingdom)

^c ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

CD11B ANTIGEN; CD18 ANTIGEN; ENHANCED GREEN FLUORESCENT PROTEIN; GANGLIOSIDE GM2; HEPARAN SULFATE; LENTIVIRUS VECTOR; PHOSPHOGLYCERATE KINASE; PROTEIN SGSH; SULFATASE; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY; B LYMPHOCYTE; BONE MARROW CELL; CELL SPECIFICITY; CODON; FEMALE; GENE OVEREXPRESSION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUMAN CELL; LYSOSOME STORAGE DISEASE; MICROGLIA; MOUSE; NEUROLOGIC DISEASE; NONHUMAN; NUCLEOTIDE SEQUENCE; PROMOTER REGION; SANFILIPPO SYNDROME TYPE A; STEM CELL GENE THERAPY; VIRAL GENE DELIVERY SYSTEM; WILD TYPE;

MUS;

ANIMALS; ANTIGENS, CD11B; BRAIN; CELL LINE; DISEASE MODELS, ANIMAL; FEMALE; GENETIC THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HEPARITIN SULFATE; HUMANS; HYDROLASES; LENTIVIRUS; LEUKOCYTES; LYSOSOMES; MICE; MICE, INBRED C57BL; MICROGLIA; MUCOPOLYSACCHARIDOSIS III; MYELOID CELLS; ORGAN SPECIFICITY; PROMOTER REGIONS, GENETIC;

EID: 84885018977     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.141     Document Type: Article

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