Therapeutic uses of inhibitors of RTP801
View Patent ↗The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases and respiratory conditions based upon inhibition of the RTP801 gene and/or protein.
1. A compound having the structure:
wherein each of A, C, G and U represents a ribonucleotide;
wherein in alternating ribonucleotides in each of the antisense and the sense strands, a 2′-O-methyl group is present in the sugar residue;
wherein the ribonucleotide at each of the 5′ terminus and the 3′ terminus of the antisense strand comprises a 2′-O-methyl group in the sugar residue;
wherein the ribonucleotide at each of the 5′ terminus and the 3′ terminus of the sense strand is unmodified in the sugar residue;
wherein each ribonucleotide at the 5′ terminus and the 3′ terminus of the antisense strand and of the sense strand is independently phosphorylated or non-phosphorylated; and
wherein each vertical line (|) represents base pairing.
2. The compound of claim 1 , wherein the ribonucleotide at the 3′ terminus of both the antisense strand and the sense strand is non-phosphorylated.
3. The compound of claim 1 , wherein in the antisense strand and in the sense strand, the ribonucleotide at the 5′ terminus and at the 3′ terminus is non-phosphorylated.
4. A pharmaceutical composition comprising a therapeutically effective amount of the compound of claim 3 and a pharmaceutically acceptable carrier or diluent.
5. A pharmaceutical composition comprising a therapeutically effective amount of the compound of claim 1 and a pharmaceutically acceptable carrier or excipient.
6. A method of treating a patient suffering from an eye disease which comprises administering to the patient by intravitreal injection the pharmaceutical composition according to claim 4 in an amount effective to treat the patient.
7. A method according to claim 6 , wherein the eye disease is macular degeneration.
8. A method according to claim 7 , wherein the macular degeneration is age-related macular degeneration (AMD).
9. A method according to claim 6 , wherein the eye disease is glaucoma.
10. A method according to claim 6 , wherein the eye disease results from laser treatment.
11. A method of treating a patient suffering from wet AMD which comprises administering to the patient by intravitreal injection a pharmaceutical composition comprising a compound having the structure:
wherein each of A, C, G and U represents a ribonucleotide;
wherein in alternating ribonucleotides in each of the antisense and the sense strands, a 2′-O-methyl group is present in the sugar residue;
wherein the ribonucleotide at each of the 5′ terminus and the 3′ terminus of the antisense strand comprises a 2′-O-methyl group in the sugar residue;
wherein the ribonucleotide at each of the 5′ terminus and the 3′ terminus of the sense strand is unmodified in the sugar residue;
wherein each vertical line (|) represents base pairing; and
wherein the pharmaceutical composition is administered in an amount effective to treat the patient.
12. The method of claim 11 , wherein in the antisense strand and in the sense strand, the ribonucleotide at the 5′ terminus and at the 3′ terminus is nonphosphorylated.