Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease

Gene Therapy

Volumn 19, Issue 11, 2012, Pages 1085-1094

  Roybal, J L ^a   Endo, M ^a   Radu, A ^a   Gray, L ^b   Todorow, C A ^a   Zoltick, P W ^a   Lutsenko, S ^b   Flake, A W ^a  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b JOHNS HOPKINS UNIVERSITY   (United States)

Author keywords

ATP7B; Copper storage disease; Lentivirus; Prenatal

Indexed keywords

BIOCHEMICAL MARKER; CERULOPLASMIN; COPPER; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE; LENTIVIRUS VECTOR; MESSENGER RNA; WILSON DISEASE PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ATP7B GENE; CONTROLLED STUDY; COPPER METABOLISM; EARLY GESTATIONAL GENE TRANSFER; FEMALE; GENE; GENE EXPRESSION; GENE THERAPY; IN UTERO GENE THERAPY; LIVER HISTOLOGY; MORPHOLOGY; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PHENOTYPE; PRIORITY JOURNAL; PROMOTER REGION; TRANSCRIPTION REGULATION; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; WILSON DISEASE;

ADENOSINE TRIPHOSPHATASES; ANIMALS; BIOLOGICAL TRANSPORT; CATION TRANSPORT PROTEINS; CERULOPLASMIN; COPPER; DISEASE MODELS, ANIMAL; FEMALE; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC THERAPY; GENETIC VECTORS; HEPATOLENTICULAR DEGENERATION; HUMANS; HYDROXYMETHYLGLUTARYL COA REDUCTASES; INJECTIONS; LIVER; LIVER FUNCTION TESTS; MALE; MICE; MICE, KNOCKOUT; ORGAN SPECIFICITY; PHENOTYPE; PROTEIN BINDING;

ANIMALIA; LENTIVIRUS; MURINAE; MUS;

EID: 84869082162     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2011.186     Document Type: Article

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