Biochemical and physiological improvement in a mouse model of Smith-Lemli-Opitz syndrome (SLOS) following gene transfer with AAV vectors

Molecular Genetics and Metabolism Reports

Volumn 1, Issue 1, 2014, Pages 103-113

  Ying, Lee ^a   Matabosch, Xavier ^a   Serra, Montserrat ^a   Watson, Berna ^a   Shackleton, Cedric ^a   Watson, Gordon ^a  

^a CHILDREN S HOSPITAL OAKLAND RESEARCH INSTITUTE   (United States)

Author keywords

7 Dehydrocholesterol reductase (DHCR7); AAV; Cholesterol synthesis; Gene therapy; Smith Lemli Opitz syndrome (SLOS)

Indexed keywords

7 DEHYDROCHOLESTEROL; 7 DEHYDROCHOLESTEROL REDUCTASE; MESSENGER RNA; OXIDOREDUCTASE; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG; CHOLESTEROL;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHOLESTEROL BLOOD LEVEL; CHOLESTEROL INTAKE; CHOLESTEROL LIVER LEVEL; CHOLESTEROL METABOLISM; CHOLESTEROL SYNTHESIS; CONTROLLED STUDY; DISEASE SEVERITY; FEMALE; GENE THERAPY; GENE TRANSFER; GROWTH RATE; INVERTED TERMINAL REPEAT; MALE; MOUSE; NEWBORN; NONHUMAN; NULL ALLELE; PROTEIN EXPRESSION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SMITH LEMLI OPITZ SYNDROME; SYSTEMIC THERAPY; VIRUS CAPSID; WEIGHT GAIN; WOODCHUCK HEPATITIS VIRUS; ADOLESCENT; EXPERIMENTAL MOUSE;

EID: 84905166394     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2014.02.002     Document Type: Article

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