Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria

Gene Therapy

Volumn 13, Issue 5, 2006, Pages 457-462

  Harding, C O ^a,b   Gillingham, M B ^b   Hamman, K ^a   Clark, H ^a   Goebel Daghighi, E ^a   Bird, A ^c   Koeberl, D D ^c  

^a OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^b OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^c DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Liver gene therapy; Mouse model; Phenylalanine; Phenylalanine hydroxylase; Phenylketonuria

Indexed keywords

COMPLEMENTARY DNA; LIVER ENZYME; PHENYLALANINE 4 MONOOXYGENASE;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; DRUG MECHANISM; DRUG SAFETY; DRUG STABILITY; ENZYME ACTIVITY; ENZYME BLOOD LEVEL; FEMALE; GENE EXPRESSION; GENETIC TRANSDUCTION; HYPERPHENYLALANINEMIA; LIVER CELL; LIVER FUNCTION; MALE; MOUSE; NONHUMAN; PARVOVIRUS VECTOR; PHENYLKETONURIA; PRIORITY JOURNAL; SEROTYPE; VIRAL GENE DELIVERY SYSTEM; VIRUS CAPSID; VIRUS RECOMBINANT; WILD TYPE;

ANIMALS; DEPENDOVIRUS; FEMALE; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HAIR COLOR; LIVER; MICE; MICE, INBRED C57BL; MODELS, ANIMAL; PHENYLALANINE HYDROXYLASE; PHENYLKETONURIAS; PIGMENTATION DISORDERS; RECOMBINANT PROTEINS; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; ANIMALIA; MIRIDAE; MURINAE; PARVOVIRUS;

EID: 33645161455     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302678     Document Type: Article

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