Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: Toxicology, biodistribution, and immunological assessments in primates

Human Gene Therapy Clinical Development

Volumn 25, Issue 2, 2014, Pages 72-84

  Murrey, Darren A ^a   Naughton, Bartholomew J ^a   Duncan, F Jason ^a   Meadows, Aaron S ^a   Ware, Tierra A ^a   Campbell, Katie J ^a   Bremer, William G ^a   Walker, Christopher M ^a,b   Goodchild, Laurie ^a   Bolon, Brad ^c   La Perle, Krista ^c   Flanigan, Kevin M ^a,b   McBride, Kim L ^a,b   McCarty, Douglas M ^a,b   Fu, Haiyan ^a,b  

^a NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b OHIO STATE UNIVERSITY   (United States)

^c OHIO STATE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA N ACETYLGLUCOSAMINIDASE; CAPSID PROTEIN; ENZYME; GAMMA INTERFERON; METHYLPREDNISOLONE; PREDNISOLONE; VIRUS ANTIBODY; VIRUS VECTOR; ACETYLGLUCOSAMINIDASE; ALPHA-N-ACETYL-D-GLUCOSAMINIDASE; ANTIBODY; RECOMBINANT PROTEIN;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS TYPE 9; ADULT; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CD4+ T LYMPHOCYTE; CELL ACTIVATION; CONTINUOUS INFUSION; CORTICOSTEROID THERAPY; DRUG ACTIVITY; DRUG SAFETY; ENZYME ACTIVITY; ENZYME BLOOD LEVEL; ENZYME LINKED IMMUNOSORBENT ASSAY; ENZYME LINKED IMMUNOSPOT ASSAY; EXPERIMENTAL MONKEY; FLOW CYTOMETRY; GENE EXPRESSION; GENETIC TRANSDUCTION; HELPER CELL; IMMUNOFLUORESCENCE; IMMUNOLOGICAL PARAMETERS; IMMUNOSUPPRESSIVE TREATMENT; MUCOPOLYSACCHARIDOSIS; MUCOPOLYSACCHARIDOSIS TYPE 3B; NONHUMAN; PRIORITY JOURNAL; QUANTITATIVE ANALYSIS; REAL TIME POLYMERASE CHAIN REACTION; SYSTEMIC THERAPY; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; ANIMAL; BLOOD; BRAIN; CENTRAL NERVOUS SYSTEM; CEREBROSPINAL FLUID; CYTOLOGY; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; IMMUNOLOGY; MACACA FASCICULARIS; METABOLISM; MUCOPOLYSACCHARIDOSIS III; TH1 CELL; TISSUE DISTRIBUTION;

ACETYLGLUCOSAMINIDASE; ANIMALS; ANTIBODIES; BRAIN; CENTRAL NERVOUS SYSTEM; DEPENDOVIRUS; ENZYME-LINKED IMMUNOSPOT ASSAY; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MACACA FASCICULARIS; MUCOPOLYSACCHARIDOSIS III; RECOMBINANT PROTEINS; TH1 CELLS; TISSUE DISTRIBUTION;

EID: 84902676801     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2013.208     Document Type: Article

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