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AAV-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells
专利权人:
Wayne State University
发明人:
Zhuo-Hua Pan
申请号:
US15236152
公开号:
US09968689B2
申请日:
2016.08.12
申请国别(地区):
US
年份:
2018
代理人:
摘要:
Microbial type rhodopsins, such as the light-gated cation-selective membrane channel, channelrhodopsin-2 (Chop2/ChR2) or the ion pump halorhodopsin (HaloR) are expressed in retinal ganglion cells upon transduction using recombinant AAV vectors. Selective targeting of these transgenes for expression in discrete subcellular regions or sites is achieved by including a sorting motif in the vector that can target either the central area or surround (off-center) area of these cells. Nucleic acid molecules comprising nucleotide sequences encoding such rhodopsins and sorting motifs and their use in methods of differential expression of the transgene are disclosed. These compositions and methods provide significant improvements for restoring visual perception and various aspects of vision, particular in patients with retinal disease.
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中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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zhuo-hua pan
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