# US 2022/0033449 A1 · Identification of channelrhodopsin-2 (chr2) mutations and methods of use

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- Number: US 2022/0033449 A1
- Document ID: US20220033449A1
- Status: Published application
- Kind: A1
- Application number: 17/202,064
- Filed: 2021-03-15
- Published: 2022-02-03
- Earliest claimed: 2012-03-05
- Assignees: Wayne State University
- Applicants: Wayne State University
- Inventors: Zhuo-Hua PAN
- Web page: https://patentgrep.com/patent/US20220033449A1

## CPC

- C07K 14/405: from algae
- A61K 38/16: Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof {(enzyme inhibitors A61K38/005)}
- A61P 27/02: Ophthalmic agents
- C07K 14/00: Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K 48/00: Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

The invention provides compositions and kits including at least one nucleic acid or polypeptide molecule encoding for a mutant ChR2 protein. Methods of the invention include administering a composition comprising a mutant ChR2 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.

## Claims

### Claim at position 1

-50. (canceled)

### Claim 51

An expression vector comprising a polynucleotide encoding a polypeptide comprising the amino acid sequence of SEQ ID NO: 26 in which the amino acid at position 132 of SEQ ID NO: 26 is cysteine (C) or alanine (A), and the amino acid at position 159 of SEQ ID NO: 26 is cysteine (C), serine (S), or alanine (A).

### Claim 52 (depends on 51)

The expression vector of claim 51, wherein the polypeptide comprises a cysteine (C) at positions 132 and 159 of SEQ ID NO: 26.

### Claim 53 (depends on 52)

The expression vector of claim 52, wherein the polypeptide comprises the amino acid sequence of SEQ ID NO: 16 and the polynucleotide comprises the nucleic acid sequence of SEQ ID NO: 15.

### Claim 54 (depends on 51)

The expression vector of claim 51, wherein the polypeptide comprises a cysteine (C) at position 132 and a serine (S) at position 159 of SEQ ID NO: 26.

### Claim 55 (depends on 54)

The expression vector of claim 54, wherein the polypeptide comprises the amino acid sequence of SEQ ID NO: 19 and the polynucleotide comprises the nucleic acid sequence of SEQ ID NO: 18.

### Claim 56 (depends on 51)

The expression vector of claim 51, wherein the polypeptide comprises a cysteine (C) at position 132 and an alanine (A) at position 159 of SEQ ID NO: 26.

### Claim 57 (depends on 56)

The expression vector of claim 56, wherein the polypeptide comprises the amino acid sequence of SEQ ID NO: 25 and the polynucleotide comprises the nucleic acid sequence of SEQ ID NO: 24.

### Claim 58 (depends on 51)

The expression vector of claim 51, wherein the polypeptide comprises an alanine (A) at position 132 and a cysteine (C) at position 159 of SEQ ID NO: 26.

### Claim 59 (depends on 58)

The expression vector of claim 58, wherein the polypeptide comprises the amino acid sequence of SEQ ID NO: 22 and the polynucleotide comprises the nucleic acid sequence of SEQ ID NO: 21.

### Claim 60 (depends on 51)

The expression vector of claim 51, wherein the polypeptide comprises an alanine (A) at position 132 and a serine (S) at position 159 of SEQ ID NO: 26.

### Claim 61 (depends on 51)

The expression vector of claim 51, wherein the polypeptide comprises an alanine (A) at position 132 and an alanine (A) at position 159 of SEQ ID NO: 26.

### Claim 62 (depends on 51)

The expression vector of claim 51, wherein the expression vector is an adeno-associated virus (AAV) vector.

### Claim 63 (depends on 62)

The expression vector of claim 62, wherein the AAV vector is an AAV2 vector.

### Claim 64 (depends on 51)

A method of improving or restoring vision in a subject, the method comprising administering to the subject the expression vector of claim 51.

### Claim 65 (depends on 62)

A method of improving or restoring vision in a subject, the method comprising administering to the subject the expression vector of claim 62.

### Claim 66 (depends on 63)

A method of improving or restoring vision in a subject, the method comprising administering to the subject the expression vector of claim 63.

### Claim 67 (depends on 64)

The method of claim 64, wherein the subject either has normal vision or the subject is suffering from an ocular disease.

### Claim 68 (depends on 65)

The method of claim 65, wherein the subject either has normal vision or the subject is suffering from an ocular disease.

### Claim 69 (depends on 66)

The method of claim 66, wherein the subject either has normal vision or the subject is suffering from an ocular disease.

### Claim 70 (depends on 67)

The method of claim 67, wherein the subject is suffering from an ocular disease and the ocular disease is macular degeneration or retinitis pigmentosa.

### Claim 71 (depends on 68)

The method of claim 68, wherein the subject is suffering from an ocular disease and the ocular disease is macular degeneration or retinitis pigmentosa.

### Claim 72 (depends on 69)

The method of claim 69, wherein the subject is suffering from an ocular disease and the ocular disease is macular degeneration or retinitis pigmentosa.