Delivery of the 7-dehydrocholesterol reductase gene to the central nervous system using adeno-associated virus vector in a mouse model of Smith-Lemli-Opitz Syndrome

Molecular Genetics and Metabolism Reports

Volumn 4, Issue , 2015, Pages 92-98

  Pasta, Saloni ^a   Akhile, Omoye ^a   Tabron, Dorothy ^a   Ting, Flora ^a   Shackleton, Cedric ^a   Watson, Gordon ^a  

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

Author keywords

7 dehydrocholesterol reductase (DHCR7); AAV; Central nervous system (CNS); Gene therapy; Intrathecal; Smith Lemli Opitz syndrome (SLOS)

Indexed keywords

7 DEHYDROCHOLESTEROL REDUCTASE; DNA; OXIDOREDUCTASE; PARVOVIRUS VECTOR; STEROL; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD BRAIN BARRIER; CENTRAL NERVOUS SYSTEM; CEREBROSPINAL FLUID; CONTROLLED STUDY; FEMALE; GENE EXPRESSION; HOMEOSTASIS; MALE; MOUSE; NEWBORN; NONHUMAN; PERIPHERAL NERVOUS SYSTEM; SMITH LEMLI OPITZ SYNDROME; VIRAL GENE DELIVERY SYSTEM;

EID: 84943616815     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2015.07.006     Document Type: Article

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