Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes
US-12173314-B2 · Dec 24, 2024 · US
US9101690B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9101690-B2 |
| Application number | US-201213555981-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jul 23, 2012 |
| Priority date | Jul 22, 2005 |
| Publication date | Aug 11, 2015 |
| Grant date | Aug 11, 2015 |
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The present invention provides compositions and methods for light-activated cation channel proteins and their uses within cell membranes and subcellular regions. The invention provides for proteins, nucleic acids, vectors and methods for genetically targeted expression of light-activated cation channels to specific cells or defined cell populations. In particular the invention provides millisecond-timescale temporal control of cation channels using moderate light intensities in cells, cell lines, transgenic animals, and humans. The invention provides for optically generating electrical spikes in nerve cells and other excitable cells useful for driving neuronal networks, drug screening, and therapy.
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What is claimed is: 1. An implantable prosthetic device comprising a cell expressing a channelrhodopsin, wherein the channelrhodopsin comprises an amino acid sequence having at least about 95% amino acid sequence identity to the amino acid sequence set forth in SEQ ID NO:1, and wherein the cell is a retinal cell, a spiral ganglion cell, or a neuron. 2. The implantable device of claim 1 , wherein the channelrhodopsin comprises an amino acid sequence having at le…
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