Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy

Molecular Therapy

Volumn 23, Issue 9, 2015, Pages 1519-1531

  Doerr, Jonas ^a   Böckenhoff, Annika ^a   Ewald, Benjamin ^a   Ladewig, Julia ^a   Eckhardt, Matthias ^a   Gieselmann, Volkmar ^a   Matzner, Ulrich ^a   Brüstle, Oliver ^a   Koch, Philipp ^a  

^a UNIVERSITY OF BONN   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CEREBROSIDE SULFATASE; KRUPPEL LIKE FACTOR 4; LENTIVIRUS VECTOR; MYC PROTEIN; OCTAMER TRANSCRIPTION FACTOR 4; SULFATIDE; TRANSCRIPTION FACTOR SOX2; DNA BINDING PROTEIN; RAG2 PROTEIN, MOUSE;

ADOLESCENT; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTE; CELL DIFFERENTIATION; CELL RENEWAL; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DISEASE MODEL; ENZYME REPLACEMENT; FEMALE; GENE OVEREXPRESSION; GENETIC ENGINEERING; HUMAN; HUMAN CELL; LIPID STORAGE; METACHROMATIC LEUKODYSTROPHY; MOUSE; NEURAL STEM CELL; NEUROEPITHELIUM CELL; NONHUMAN; NUCLEAR REPROGRAMMING; PLURIPOTENT STEM CELL; STEM CELL TRANSPLANTATION; TREATMENT OUTCOME; ANIMAL; AXON; BIOLOGICAL THERAPY; BRAIN; CELL SURVIVAL; CYTOLOGY; DEFICIENCY; GENE EXPRESSION; GENE ORDER; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; GLIA; INDUCED PLURIPOTENT STEM CELL; KNOCKOUT MOUSE; LENTIVIRINAE; METABOLISM; NERVE CELL; PROCEDURES;

ANIMALS; AXONS; BRAIN; CELL DIFFERENTIATION; CELL SURVIVAL; CELL- AND TISSUE-BASED THERAPY; CENTRAL NERVOUS SYSTEM; CEREBROSIDE-SULFATASE; DISEASE MODELS, ANIMAL; DNA-BINDING PROTEINS; GENE EXPRESSION; GENE ORDER; GENETIC THERAPY; GENETIC VECTORS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; LENTIVIRUS; LEUKODYSTROPHY, METACHROMATIC; MICE; MICE, KNOCKOUT; NEUROGLIA; NEURONS; SULFOGLYCOSPHINGOLIPIDS; TRANSDUCTION, GENETIC;

EID: 84940097054     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.106     Document Type: Article

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