Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer

Molecular Therapy

Volumn 16, Issue 3, 2008, Pages 458-465

  Bennicelli, Jeannette ^a   Wright, John Fraser ^b,c   Komaromy, Andras ^d   Jacobs, Jonathan B ^e   Hauck, Bernd ^b   Zelenaia, Olga ^b   Mingozzi, Federico ^b   Hui, Daniel ^b   Chung, Daniel ^a   Rex, Tonia S ^a   Wei, Zhangyong ^a   Qu, Guang ^b   Zhou, Shangzhen ^b   Zeiss, Caroline ^f   Arruda, Valder R ^b   Acland, Gregory M ^g   Dell'Osso, Lou F ^e   High, Katherine A ^b   Maguire, Albert M ^a   Bennett, Jean ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

^d UNIVERSITY OF PENNSYLVANIA SCHOOL OF VETERINARY MEDICINE   (United States)

^e CASE WESTERN RESERVE UNIVERSITY   (United States)

^f YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^g Animal Health Diagnostic Center   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EYE PROTEIN; PARVOVIRUS VECTOR; RETINA PIGMENT EPITHELIUM PROTEIN 65; SURFACTANT; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLINDNESS; CONTROLLED STUDY; DOG; ELECTRORETINOGRAPHY; EXPERIMENTAL ANIMAL; GENE MUTATION; GENE SEQUENCE; GENE STRUCTURE; IMMUNOHISTOCHEMISTRY; KOZAK SEQUENCE; LEBER CONGENITAL AMAUROSIS; MOUSE; MUTANT; NONHUMAN; NYSTAGMUS; OUTCOME ASSESSMENT; PIGMENT EPITHELIUM; PROTEIN EXPRESSION; TRANSLATION INITIATION; VIRAL GENE DELIVERY SYSTEM; VISUAL ACUITY;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CANIS FAMILIARIS; MUS;

EID: 39849110435     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300389     Document Type: Article

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