发明名称 RESTORATION OF VISUAL RESPONSES BY IN VIVO DELIVERY OF RHODOPSIN NUCLEIC ACIDS
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.
申请公布号 EP2019588(A4) 申请公布日期 2010.11.24
申请号 EP20070797340 申请日期 2007.05.04
申请人 WAYNE STATE UNIVERSITY;PENNSYLVANIA COLLEGE OF OPTOMETRY 发明人 PAN, ZHUO-HUA;DIZHOOR, ALEXANDER, M.
分类号 A01N43/04;C07K14/705;C12N15/00 主分类号 A01N43/04
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