Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

Proceedings of the National Academy of Sciences of the United States of America

Volumn 109, Issue 6, 2012, Pages 2132-2137

  Beltran, William A ^a   Cideciyan, Artur V ^b   Lewin, Alfred S ^c   Iwabe, Simone ^a   Khanna, Hemant ^d,e   Sumaroka, Alexander ^b   Chiodo, Vince A ^f   Fajardo, Diego S ^c   Román, Alejandro J ^b   Deng, Wen Tao ^f   Swider, Malgorzata ^b   Alemán, Tomas S ^b   Boye, Sanford L ^f   Genini, Sem ^a   Swaroop, Anand ^d,g   Hauswirth, William W ^f   Jacobson, Samuel G ^b   Aguirre, Gustavo D ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c UNIVERSITY OF FLORIDA   (United States)

^d UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^e UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

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

^g NATIONAL EYE INSTITUTE   (United States)

Author keywords

Retina; Retinal degeneration

Indexed keywords

COMPLEMENTARY DNA; GUANOSINE TRIPHOSPHATASE; OPSIN; PROTEIN;

ADENO ASSOCIATED VIRUS; ADULT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLINDNESS; CHILD; CLINICAL EFFECTIVENESS; CONTROLLED STUDY; DENDRITE; DOG; ELECTRORETINOGRAPHY; GENE MUTATION; GENE THERAPY; GENE TRANSFER; GENOTYPE; HISTOPATHOLOGY; HUMAN; IN VIVO STUDY; MORPHOLOGY; NONHUMAN; OPTICAL COHERENCE TOMOGRAPHY; OUTCOME ASSESSMENT; PHENOTYPE; PHOTORECEPTOR; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; RETINA BIPOLAR GANGLION CELL; RETINA CONE; RETINA INNER PLEXIFORM LAYER; RETINA ROD; RETINITIS PIGMENTOSA; SCHOOL CHILD;

ANIMALS; BLINDNESS; DOGS; EYE PROTEINS; GENE THERAPY; GENETIC DISEASES, X-LINKED; GENOTYPE; HUMANS; MICE; MUTATION; OPEN READING FRAMES; OPSINS; PHENOTYPE; PHOTORECEPTOR CELLS, VERTEBRATE; PROTEIN TRANSPORT; RETINAL BIPOLAR CELLS; RETINITIS PIGMENTOSA;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CANIS FAMILIARIS;

EID: 84857129967     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1118847109     Document Type: Article

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