A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB

Human Gene Therapy Clinical Development

Volumn 26, Issue 4, 2015, Pages 228-242

  Meadows, Aaron S ^a   Duncan, F Jason ^a   Camboni, Marybeth ^a   Waligura, Kathryn ^a   Montgomery, Chrystal ^a   Zaraspe, Kimberly ^a   Naughton, Bartholomew J ^a   Bremer, William G ^a   Shilling, Christopher ^a   Walker, Christopher M ^a,b   Bolon, Brad ^c   Flanigan, Kevin M ^a,b   McBride, Kim L ^b,d   McCarty, Douglas M ^a,b   Fu, Haiyan ^a  

^a THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b Immunology and Medical Genetics   (United States)

^c OHIO STATE UNIVERSITY   (United States)

^d Center for Cardiovascular and Pulmonary Research   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ALKALINE PHOSPHATASE; ALPHA N ACETYLGLUCOSAMINIDASE; ASPARTATE AMINOTRANSFERASE; BILIRUBIN; CREATINE KINASE; CREATININE; GAMMA GLUTAMYLTRANSFERASE; GAMMA INTERFERON; IMMUNOGLOBULIN G; NITROGEN; PARVOVIRUS VECTOR; PROTEIN; RECOMBINANT ADENOASSOCIATED VIRUS 9 VECTOR; SODIUM CHLORIDE; TRIACYLGLYCEROL; UNCLASSIFIED DRUG; UREA; ACETYLGLUCOSAMINIDASE; ALPHA-N-ACETYL-D-GLUCOSAMINIDASE;

ACUTE TOXICITY; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ALKALINE PHOSPHATASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY RESPONSE; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; AUTOPSY; BILIRUBIN BLOOD LEVEL; BLOOD CHEMISTRY; BLOOD SAMPLING; BODY WEIGHT; CARDIOMYOPATHY; CELL INFILTRATION; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; CREATININE BLOOD LEVEL; DOSE RESPONSE; ENZYME ACTIVITY; ENZYME LINKED IMMUNOSORBENT ASSAY; FEMALE; GAMMA GLUTAMYL TRANSFERASE BLOOD LEVEL; GENE EXPRESSION; GENE TARGETING; GENOTYPE; HISTOPATHOLOGY; IMMUNOFLUORESCENCE TEST; IMMUNOGENICITY; LIVER NECROSIS; LIVER TOXICITY; LYSOSOME STORAGE DISEASE; MALE; MICROSCOPY; MORBIDITY; MOUSE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PALLOR; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; PROTEIN EXPRESSION; TRIACYLGLYCEROL BLOOD LEVEL; UREA NITROGEN BLOOD LEVEL; VIRAL GENE DELIVERY SYSTEM; ADMINISTRATION AND DOSAGE; ADVERSE EFFECTS; ANIMAL; ANTIBODY SPECIFICITY; BRAIN; C57BL MOUSE; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; LIVER; METABOLISM; MUCOPOLYSACCHARIDOSIS III; PRACTICE GUIDELINE; PROCEDURES; SPINAL CORD;

ACETYLGLUCOSAMINIDASE; ANIMALS; BRAIN; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; LIVER; MALE; MICE; MICE, INBRED C57BL; MUCOPOLYSACCHARIDOSIS III; ORGAN SPECIFICITY; PRACTICE GUIDELINES AS TOPIC; SPINAL CORD;

EID: 85006219537     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2015.132     Document Type: Article

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