Invention Grant
- Patent Title: AAV-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells
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Application No.: US15236152Application Date: 2016-08-12
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Publication No.: US09968689B2Publication Date: 2018-05-15
- Inventor: Zhuo-Hua Pan
- Applicant: Wayne State University
- Applicant Address: US MI Detroit
- Assignee: WAYNE STATE UNIVERSITY
- Current Assignee: WAYNE STATE UNIVERSITY
- Current Assignee Address: US MI Detroit
- Agent Cynthia A. Kozakiewicz; Ivor Elrifi
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C07K14/47 ; C07K14/705 ; C12N15/861 ; A61K38/00

Abstract:
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.
Public/Granted literature
- US20160354488A1 AAV-Mediated Subcellular Targeting of Heterologous Rhodopsins in Retinal Ganglion Cells Public/Granted day:2016-12-08
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