Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis

Molecular Therapy

Volumn 20, Issue 2, 2012, Pages 267-274

  Hu, Huimin ^a   Gomero, Elida ^a   Bonten, Erik ^a   Gray, John T ^a   Allay, Jim ^a   Wu, Yanan ^a   Wu, Jianrong ^a   Calabrese, Christopher ^a   Nienhuis, Arthur ^a   D'Azzo, Alessandra ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CARBOXYPEPTIDASE C; GUANOSINE PHOSPHATE; PARVOVIRUS VECTOR; PROTECTIVE PROTEIN CATHEPSIN A; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS; AGED; ANIMAL BEHAVIOR; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; COHORT ANALYSIS; CONTROLLED STUDY; DOSE RESPONSE; DRUG DISTRIBUTION; DRUG DOSE COMPARISON; DRUG DOSE REGIMEN; DRUG MEGADOSE; DRUG SAFETY; DRUG SCREENING; FEMALE; FEMALE INFERTILITY; GALACTOSIALIDOSIS; GENITAL SYSTEM; HUMAN; HUMAN CELL; INFANT; LIVER; LOW DRUG DOSE; MALE; MALE INFERTILITY; MOUSE; NONHUMAN; PHYSICAL EXAMINATION; PROMOTER REGION; PROTEIN EXPRESSION; SERUM; TISSUE DISTRIBUTION; TISSUE SPECIFICITY; TRANSGENE; URINE; VIRAL GENE DELIVERY SYSTEM; VIRUS EXPRESSION; VIRUS RECOMBINANT; VIRUS TYPING;

MUS;

EID: 84856529857     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.227     Document Type: Article

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