TREATMENT OF EYE DISEASE
A method of treating an eye disease comprising administering an adeno-associated virus (AAV) vector to a mammalian subject by subretinal injection, wherein the AAV vector comprises a nucleotide sequence encoding melanopsin operably linked to an expression control sequence to promote expression of melanopsin in cells of the eye of the subject.
1 . A method of treating an eye disease comprising administering an adeno-associated virus (AAV) vector to a mammalian subject by subretinal injection, wherein the AAV vector comprises a nucleotide sequence encoding melanopsin operably linked to an expression control sequence to promote expression of melanopsin in cells of the eye of the subject.
2 . The method of claim 1 , wherein the eye disease is a retinal dystrophy.
3 . The method of claim 1 , wherein the eye disease is retinitis pigmentosa.
4 . The method of any preceding claim, wherein the eye to be treated lacks rod and/or cone photoreceptor cells.
5 . The method of any preceding claim, wherein the AAV vector is in the form of an AAV particle comprising an AAV8 Y733F mutant capsid.
6 . The method of any preceding claim, wherein the AAV vector comprises an AAV2 genome.
7 . The method of any preceding claim, wherein the melanopsin is expressed in bipolar and/or horizontal cells.
8 . The method of any preceding claim, wherein the melanopsin is human melanopsin.
9 . The method of any preceding claim, wherein the expression control sequence comprises a CBA promoter.
10 . The method of any preceding claim, wherein the subretinal injection comprises the steps:
(a) administering a solution to the subject by subretinal injection in an amount effective to at least partially detach the retina to form a subretinal bleb, wherein the solution does not comprise the AAV vector; and
(b) administering a medicament composition by subretinal injection into the bleb formed by step (a), wherein the medicament comprises the AAV vector.
11 . A method of improving or restoring vision comprising administering an adeno-associated virus (AAV) vector to a mammalian subject by subretinal injection, wherein the AAV vector comprises a nucleotide sequence encoding melanopsin operably linked to an expression control sequence to promote expression of melanopsin in cells of the eye of the subject.
12 . The method of claim 11 , wherein the subject suffers from a retinal dystrophy.
13 . The method of claim 11 , wherein the subject suffers from retinitis pigmentosa.
14 . The method of any of claims 11 - 13 , wherein the eye to be treated lacks rod and/or cone photoreceptor cells.
15 . The method of any of claims 11 - 14 , wherein the AAV vector is in the form of an AAV particle comprising an AAV8 Y733F mutant capsid.
16 . The method of any of claims 11 - 15 , wherein the AAV vector comprises an AAV2 genome.
17 . The method of any of claims 11 - 16 , wherein the melanopsin is expressed in bipolar and/or horizontal cells.
18 . The method of any of claims 11 - 17 , wherein the melanopsin is human melanopsin.
19 . The method of any of claims 11 - 18 , wherein the expression control sequence comprises a CBA promoter.
20 . The method of any of claims 11 - 19 , wherein the subretinal injection comprises the steps:
(a) administering a solution to the subject by subretinal injection in an amount effective to at least partially detach the retina to form a subretinal bleb, wherein the solution does not comprise the AAV vector; and
(1) administering a medicament composition by subretinal injection into the bleb formed by step (a), wherein the medicament comprises the AAV vector.