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1. WO2016149664 - METHODS AND COMPOSITIONS FOR RESTORATION OF CONE FUNCTION IN BCM

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[ EN ]

CLAIMS

1. A nucleic acid comprising an expression construct containing an M-opsin gene operably linked to a cone-specific promoter, wherein the expression construct is flanked by inverted terminal repeat sequences.

2. The nucleic acid of claim 1, wherein the cone-specific promoter is a PR2.1 promoter having the sequence of SEQ ID NO: 1.

3. The nucleic acid of claim 1 or claim 2, wherein the expression construct further contains nucleic acid segments that encode an SV40 polyadenylation signal and a bovine growth hormone polyadenylation signal, wherein the nucleic acid segments are positioned 3' to the M-opsin gene.

4. The nucleic acid of any one of claims 1 to 3, wherein the nucleic acid comprises the sequence of SEQ ID NO: 2.

5. The nucleic acid of any one of claims 1 to 4, wherein the nucleic acid is a recombinant adeno-associated virus (rAAV) vector.

6. A recombinant adeno-associated virus (rAAV) particle comprising the nucleic acid of claim 5.

7. The rAAV particle of claim 6, wherein the rAAV particle is an AAV5 particle.

8. A composition comprising a plurality of the rAAV particle of claim 6 or claim 7.

9. The composition of claim 8, further comprising a pharmaceutically acceptable carrier.

10. A method of treating a cone monochromacy in a subject, the method comprising: administering a therapeutically effective amount of the rAAV particle of claim 6 or claim 7 or the composition of claim 8 or claim 9 to a subject having a cone monochromacy.

11. The method of claim 10, wherein the rAAV particle or composition are administered via subretinal injection.

12. The method of claim 10 or claim 11, wherein the subject has blue cone

monochromacy.