Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

Molecular Therapy

Volumn 26, Issue 4, 2018, Pages 1127-1136

  Laoharawee, Kanut ^a   DeKelver, Russell C ^b   Podetz Pedersen, Kelly M ^a   Rohde, Michelle ^b   Sproul, Scott ^b   Nguyen, Hoang Oanh ^b   Nguyen, Tam ^a   St Martin, Susan J ^b   Ou, Li ^a   Tom, Susan ^b   Radeke, Robert ^b   Meyer, Kathleen E ^b   Holmes, Michael C ^b   Whitley, Chester B ^a   Wechsler, Thomas ^b   McIvor, R Scott ^a  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b SANGAMO BIOSCIENCES INC   (United States)

Author keywords

albumin locus; gene therapy; Hunter syndrome; iduronate 2 sulfatase; in vivo genome editing; lysosomal disease; MPS II; zinc finger nuclease

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALBUMIN; GLYCOSAMINOGLYCAN; IDURONATE 2 SULFATASE; ZINC FINGER NUCLEASE; BIOLOGICAL MARKER; ENDONUCLEASE; ZINC FINGER PROTEIN;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; FEMALE; GENE EDITING; GENE INSERTION; GENE LOCUS; HUNTER SYNDROME; IN VIVO STUDY; INTRON; MALE; METABOLIC DISORDER; MOUSE; MOUSE MODEL; NEUROLOGIC DISEASE; NONHUMAN; PROTEIN EXPRESSION; ANIMAL; DISEASE MODEL; ENERGY METABOLISM; ENZYME ACTIVATION; GENE DOSAGE; GENE TRANSFER; GENETICS; LIVER CELL; METABOLISM; PATHOLOGY; PATHOPHYSIOLOGY; PHENOTYPE;

ANIMALS; BIOMARKERS; DISEASE MODELS, ANIMAL; ENDONUCLEASES; ENERGY METABOLISM; ENZYME ACTIVATION; GENE DOSAGE; GENE EDITING; GENE TRANSFER TECHNIQUES; HEPATOCYTES; IDURONATE SULFATASE; INTRONS; MICE; MUCOPOLYSACCHARIDOSIS II; PHENOTYPE; ZINC FINGERS;

EID: 85044263291     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2018.03.002     Document Type: Article

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