Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

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

Volumn 112, Issue 43, 2015, Pages E5844-E5853

  Beltran, William A ^a   Cideciyan, Artur V ^a   Iwabe, Simone ^a   Swider, Malgorzata ^b   Kosyk, Mychajlo S ^b   McDaid, Kendra ^a   Martynyuk, Inna ^a   Ying, Gui Shuang ^b   Shaffer, James ^b   Deng, Wen Tao ^c   Boye, Sanford L ^c   Lewin, Alfred S ^d   Hauswirth, William W ^c   Jacobson, Samuel G ^b   Aguirre, Gustavo D ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

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

^d UNIVERSITY OF FLORIDA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; GUANOSINE TRIPHOSPHATASE; GUANOSINE TRIPHOSPHATASE REGULATOR PROTEIN; OPSIN; PARVOVIRUS VECTOR; REGULATOR PROTEIN; UNCLASSIFIED DRUG;

ANIMAL MODEL; ARTICLE; CANINE MODEL; CONTROLLED STUDY; DISEASE COURSE; DRUG EFFICACY; GENE TRANSFER; HISTOLOGY; HUMAN; IN VIVO STUDY; NONHUMAN; PHOTORECEPTOR; PRIORITY JOURNAL; RETINA DEGENERATION; RETINITIS PIGMENTOSA; SPATIOTEMPORAL ANALYSIS; SUBRETINAL DRUG ADMINISTRATION; TRANSGENE; VIRAL GENE THERAPY; VISUAL IMPAIRMENT; X CHROMOSOME LINKED DISORDER; X LINKED RETINITIS PIGMENTOSA; ANIMAL; DISEASE MODEL; DOG; GENE THERAPY; METABOLISM; PATHOPHYSIOLOGY; PHOTORECEPTOR CELL; RETINA; RETINAL DEGENERATION; VISION;

ANIMALS; DISEASE MODELS, ANIMAL; DOGS; GENETIC THERAPY; PHOTORECEPTOR CELLS, VERTEBRATE; RETINA; RETINAL DEGENERATION; VISION, OCULAR;

EID: 84945338029     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1509914112     Document Type: Article

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