Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy

Molecular Therapy

Volumn 18, Issue 11, 2010, Pages 1983-1994

  Hurlbut, Gregory D ^a   Ziegler, Robin J ^a   Nietupski, Jennifer B ^a   Foley, Joseph W ^a   Woodworth, Lisa A ^a   Meyers, Elizabeth ^a   Bercury, Scott D ^a   Pande, Nilesh N ^a   Souza, David W ^a   Bree, Mark P ^a   Lukason, Michael J ^a   Marshall, John ^a   Cheng, Seng H ^a   Scheule, Ronald K ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GALACTOSIDASE; PARVOVIRUS VECTOR; RNA; VIRUS ANTIBODY;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY DETECTION; ANTIBODY TITER; ARTICLE; CONTROLLED STUDY; DISEASE MODEL; GENE EXPRESSION; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; IMMUNE RESPONSE; LYSOSOME STORAGE DISEASE; MALE; MOUSE; NONHUMAN; PASSIVE TRANSPORT; PREDICTION; PRIMATE; QUANTITATIVE ANALYSIS; RNA TRANSLATION; TRANSGENE; VIRAL GENE THERAPY;

ADENO-ASSOCIATED VIRUS; MUS; PRIMATES;

EID: 78049478714     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.175     Document Type: Article

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