| 摘要 |
PROBLEM TO BE SOLVED: To provide restoration of a visual response by in vivo delivery of a rhodopsin nucleic acid.SOLUTION: A nucleic acid vector encoding a light-gated cation-selective membrane channel, in particular channelrhodopsin-2 (Chop2), converts an inner retinal neuron to a photosensitive cell in a photoreceptor-degenerated retina in an animal model. Such treatment restores 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 a step of 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 a transcriptional regulatory sequence, so that the nucleic acid is expressed in the inner retinal neuron.SELECTED DRAWING: Figure 7 |
