Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis

Human Molecular Genetics

Volumn 20, Issue 23, 2011, Pages 4569-4581

  Ku, Cristy A ^a   Chiodo, Vince A ^b   Boye, Sanford L ^b   Goldberg, Andrew F X ^c   Li, Tiansen ^d   Hauswirth, William W ^b   Ramamurthy, Visvanathan ^a  

^a WEST VIRGINIA UNIVERSITY   (United States)

^b UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^c OAKLAND UNIVERSITY   (United States)

^d NATIONAL EYE INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; AROMATIC HYDROCARBON RECEPTOR; ARYL HYDROCARBON RECEPTOR INTERACTING PROTEIN LIKE 1; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; GENE THERAPY; HUMAN; LEBER CONGENITAL AMAUROSIS; MOUSE; NONHUMAN; PRIORITY JOURNAL; RETINA DEGENERATION;

ADAPTOR PROTEINS, SIGNAL TRANSDUCING; ANIMALS; BEHAVIOR, ANIMAL; CAPSID; CAPSID PROTEINS; CARRIER PROTEINS; CYCLIC NUCLEOTIDE PHOSPHODIESTERASES, TYPE 6; DEPENDOVIRUS; EYE PROTEINS; GENE THERAPY; HUMANS; LEBER CONGENITAL AMAUROSIS; LIGHT; MICE; MUTATION; RETINA; RETINAL DEGENERATION; RETINAL ROD PHOTORECEPTOR CELLS; VISION, OCULAR;

MIRIDAE; MUS;

EID: 81255169334     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddr391     Document Type: Article

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