Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof
AssigneeWayne State University
InventorsZhuo-Hua Pan, Tushar Ganjawala, Qi Lu, Gary Abrams
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Abstract
The invention provides compositions and kits including at least one nucleic acid or polypeptide molecule encoding for a mutant CoChop protein. Methods of the invention include administering a composition comprising a mutant CoChop to a subject to preserve, improve, or restore phototransduction. Preferably, the compositions and methods of the invention are provided to a subject having impaired vision, thereby restoring vision to normal levels.
Claims23
A method of treating Stargardt disease in a subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a nucleic acid encoding a polypeptide comprising the amino acid sequence of SEQ ID NO:
- 10, wherein the nucleic acid is operably linked to a promoter and the polypeptide is expressed in a cell membrane of a retinal neuron of the subject following administration.
A method of treating geographic atrophy in a subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a nucleic acid encoding a polypeptide comprising the amino acid sequence of SEQ ID NO:
- 10, wherein the nucleic acid is operably linked to a promoter and the polypeptide is expressed in a cell membrane of a retinal neuron of the subject following administration.
A method of treating macular degeneration in a subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a nucleic acid encoding a polypeptide comprising the amino acid sequence of SEQ ID NO:
- 10, wherein the nucleic acid is operably linked to a promoter and the polypeptide is expressed in a cell membrane of a retinal neuron of the subject following administration.
Dependencies are inferred from the claim text.