Adenoviral-mediated expression of porphobilinogen deaminase in liver restores the metabolic defect in a mouse model of acute intermittent porphyria

Molecular Therapy

Volumn 10, Issue 2, 2004, Pages 337-343

  Johansson, Annika ^a   Nowak, Grzegorz ^b   Möller, Christer ^c   Blomberg, Pontus ^b   Harper, Pauline ^a  

^a Division of Clinical Chemistry   (Sweden)

^b KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

^c HemeBiotech A/S   (Sweden)

Author keywords

5 aminolevulinic acid; Acute intermittent porphyria; Porphobilinogen; Porphobilinogen deaminase; Recombinant adenoviral vector

Indexed keywords

ADENOVIRUS VECTOR; AMINOLEVULINIC ACID; COMPLEMENTARY DNA; HEME; LUCIFERASE; PHENOBARBITAL; PORPHOBILINOGEN; PORPHOBILINOGEN DEAMINASE; PORPHYRIN;

ACUTE INTERMITTENT PORPHYRIA; AMINO ACID BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DOSE TIME EFFECT RELATION; ENZYME ACTIVITY; ENZYME DEFICIENCY; GENE EXPRESSION; HEME SYNTHESIS; HUMAN; HUMAN CELL; IN VIVO STUDY; KIDNEY; LIVER; LIVER LEVEL; MOUSE; NONHUMAN; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; WILD TYPE;

ADENOVIRIDAE; AMINOLEVULINIC ACID; ANIMALS; DISEASE MODELS, ANIMAL; GENE EXPRESSION; GENE THERAPY; HYDROXYMETHYLBILANE SYNTHASE; LIVER; LUCIFERASES; MICE; MICE, MUTANT STRAINS; PHENOBARBITAL; PORPHYRIA, ACUTE INTERMITTENT; TISSUE DISTRIBUTION;

EID: 4344700407     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.05.018     Document Type: Article

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