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US Patent 9453241 AAV-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells

Patent 9453241 was granted and assigned to Wayne State University on September, 2016 by the United States Patent and Trademark Office.

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Current Assignee
Wayne State University
Wayne State University
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Patent Jurisdiction
United States Patent and Trademark Office
United States Patent and Trademark Office
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Patent Number
94532410
Patent Inventor Names
Zhuo-Hua Pan0
Date of Patent
September 27, 2016
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Patent Application Number
136962520
Date Filed
May 4, 2011
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Patent Citations Received
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US Patent 11905523 Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
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US Patent 12123013 WPRE mutant constructs, compositions, and methods thereof
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US Patent 12077783 AAV-mediated delivery of antibodies to the inner ear
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US Patent 12071633 Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
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Patent Primary Examiner
Janet Epps-Smith
Janet Epps-Smith
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Patent abstract

Microbial type rhodopsins, such as the light-gated cation-selective membrane channel, channel-rhodopsin-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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