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Restoration of Visual Responses by In Vivo Delivery of Rhodopsin Nucleic Acids
专利权人:
Wayne State University;Salus University
发明人:
Zhuo-Hua PAN,Alexander M. DIZHOOR
申请号:
US15676573
公开号:
US20180214513A1
申请日:
2017.08.14
申请国别(地区):
US
年份:
2018
代理人:
摘要:
Nucleic acid vectors encoding light-gated cation-selective membrane channels, in particular channelrhodopsin-2 (Chop2), converted inner retinal neurons to photosensitive cells in photoreceptor-degenerated retina in an animal model. Such treatment restored visual perception and various aspects of vision. A method of restoring light sensitivity to a retina of a subject suffering from vision loss due to photoreceptor degeneration, as in retinitis pigmentosa or macular degeneration, is provided. The method comprises delivering to the subject by intravitreal or subretinal injection, the above nucleic acid vector which comprises an open reading frame encoding a rhodopsin, to which is operatively linked a promoter and transcriptional regulatory sequences, so that the nucleic acid is expressed in inner retinal neurons. These cells, normally light-insensitive, are converted to a light-sensitive state and transmit visual information to the brain, compensating for the loss, and leading to restoration of various visual capabilities.
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