RNA interference in ocular indications
First Claim
Patent Images
1. A method for delivering a nucleic acid to an eye of a subject in need thereof, comprisingadministering to the eye of the subject an sd-rxRNA®
- , in an effective amount to promote RNA interference by the sd-rxRNA®
in the eye,wherein the sd-rxRNA®
comprises a guide strand and a passenger strand, wherein the sd-rxRNA®
includes a double stranded region and a single stranded region, wherein the double stranded region is 8-15 nucleotides long, wherein the single stranded region is at the 3′
end of the guide strand and is 4-12 nucleotides long, wherein the single stranded region contains 3, 4, 5, 6, 7, 8, 9, 10, 11, or 12 phosphorothioate modifications, wherein at least 40% of the nucleotides of the sd-rxRNA®
are modified,wherein the sd-rxRNA®
is directed against a gene encoding TGFβ
1 or TGFβ
2, and wherein the sd-rxRNA comprises at least 12 contiguous nucleotides of a sequence selected from the group consisting of SEQ ID NOs;
1027-1314.
3 Assignments
0 Petitions
Accused Products
Abstract
The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.
356 Citations
19 Claims
-
1. A method for delivering a nucleic acid to an eye of a subject in need thereof, comprising
administering to the eye of the subject an sd-rxRNA® - , in an effective amount to promote RNA interference by the sd-rxRNA®
in the eye,wherein the sd-rxRNA®
comprises a guide strand and a passenger strand, wherein the sd-rxRNA®
includes a double stranded region and a single stranded region, wherein the double stranded region is 8-15 nucleotides long, wherein the single stranded region is at the 3′
end of the guide strand and is 4-12 nucleotides long, wherein the single stranded region contains 3, 4, 5, 6, 7, 8, 9, 10, 11, or 12 phosphorothioate modifications, wherein at least 40% of the nucleotides of the sd-rxRNA®
are modified,wherein the sd-rxRNA®
is directed against a gene encoding TGFβ
1 or TGFβ
2, and wherein the sd-rxRNA comprises at least 12 contiguous nucleotides of a sequence selected from the group consisting of SEQ ID NOs;
1027-1314. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19)
- , in an effective amount to promote RNA interference by the sd-rxRNA®
Specification