Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency

Gene Therapy

Volumn 17, Issue 7, 2010, Pages 815-826

  Pang, J ^a   Boye, S E ^a   Lei, B ^b,c   Boye, S L ^a   Everhart, D ^d   Ryals, R ^a   Umino, Y ^d   Rohrer, B ^e   Alexander, J ^f   Li, J ^a   Dai, X ^a,g   Li, Q ^a   Chang, B ^h   Barlow, R ^d   Hauswirth, W W ^a  

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

^b University of Missouri   (United States)

^c THE FIRST AFFILIATED HOSPITAL OF CHONGQING MEDICAL UNIVERSITY   (China)

^d State University of New York Upstate Medical University: College of Medicine   (United States)

^e MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^f UNIVERSITY OF FLORIDA   (United States)

^g WENZHOU MEDICAL UNIVERSITY   (China)

^h JACKSON LABORATORY   (United States)

Author keywords

adeno associated virus; Leber's congenital amaurosis 2; rd12 mice; retinal gene therapy; RPE65; Rpe65 :Rho mice

Indexed keywords

PARVOVIRUS VECTOR; PROTEIN P14; PROTEIN P35; RHODOPSIN; CARRIER PROTEIN; CIS TRANS ISOMERASE; EYE PROTEIN; RETINOID ISOMEROHYDROLASE;

ADENO ASSOCIATED VIRUS; ADENOVIRUS 5; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; ELECTRORETINOGRAM; GENE EXPRESSION; HISTOPATHOLOGY; MOUSE; NONHUMAN; PERINATAL PERIOD; PIGMENT EPITHELIUM; PRIORITY JOURNAL; PROTEIN EXPRESSION; RETINA CONE; RETINA PIGMENT DEGENERATION; RETINA ROD; VIRAL GENE THERAPY; VISION; VISUAL IMPAIRMENT; ANIMAL; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; OPTIC ATROPHY, HEREDITARY, LEBER; PHYSIOLOGY; RETINAL DEGENERATION;

ANIMALS; CARRIER PROTEINS; CIS-TRANS-ISOMERASES; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; EYE PROTEINS; GENETIC THERAPY; GENETIC VECTORS; MICE; OPTIC ATROPHY, HEREDITARY, LEBER; RETINAL CONE PHOTORECEPTOR CELLS; RETINAL DEGENERATION;

EID: 77954493492     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2010.29     Document Type: Article

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