RNA interference in ocular indications
First Claim
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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 from 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 isolated double stranded nucleic acid molecule are modified,wherein the sd-rxRNA®
is directed against a gene encoding VEGF or CTGF, andwherein the sd-rxRNA®
comprises at least 12 contiguous nucleotides of a sequence selected from the sequences within Tables 5, 8 or 10.
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Abstract
The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.
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Citations
10 Claims
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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 from 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 isolated double stranded nucleic acid molecule are modified,wherein the sd-rxRNA®
is directed against a gene encoding VEGF or CTGF, andwherein the sd-rxRNA®
comprises at least 12 contiguous nucleotides of a sequence selected from the sequences within Tables 5, 8 or 10. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10)
- , in an effective amount to promote RNA interference by the sd-rxRNA®
Specification