US2012108647A1PendingUtilityA1

Therapeutic uses of inhibitors of rtp801l

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Assignee: FEINSTEIN ELENAPriority: Jun 9, 2006Filed: Aug 25, 2011Published: May 3, 2012
Est. expiryJun 9, 2026(expired)· nominal 20-yr term from priority
A61P 9/00A61P 25/00A61P 27/02A61P 27/06A61P 27/00C12N 15/113C12N 2310/321C12N 2320/31C12N 2310/14A61P 11/00A61P 11/06
56
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Claims

Abstract

The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases respiratory conditions and hearing disorders based upon inhibition of the RTP801L gene and/or protein.

Claims

exact text as granted — not AI-modified
1 .- 48 . (canceled) 
     
     
         49 . A method of treating a patient suffering from an eye disease or disorder comprising administering to the patient a therapeutically effective dose of a RTP801L inhibitor so as to thereby treat the patient. 
     
     
         50 . The method of  claim 49  wherein the eye disease comprises glaucoma. 
     
     
         51 . The method of  claim 49  wherein the eye disease comprises acute retinal necrosis 
     
     
         52 . The method of  claim 49  wherein the RTP801L inhibitor is administered to the eye topically or in the form of an intravitreal injection. 
     
     
         53 . The method of  claim 49  wherein the RTP801L inhibitor comprises a polynucleotide which specifically hybridizes to a mRNA transcribed from a RTP801L gene and down regulates the expression of the RTP801L gene. 
     
     
         54 . The method of  claim 53  wherein the polynucleotide is a siRNA. 
     
     
         55 . The method of  claim 49  wherein the RTP801L inhibitor is a compound having the structure 
       
         
           
           
               
               
           
         
         wherein alternating ribonucleotides in the antisense strand and the sense strand are 2′-O-methyl sugar modified ribonucleotides; wherein the ribonucleotides at the 5′ terminus and the 3′ terminus of the antisense strand are 2′-O-methyl sugar modified; and wherein the ribonucleotides at the 5′ terminus and the 3′ terminus of the sense strand are unmodified. 
       
     
     
         56 . The method of  claim 49  wherein the RTP801L inhibitor is a compound having the structure 
       
         
           
           
               
               
           
         
         wherein alternating ribonucleotides in the antisense strand and the sense strand are 2′-O-methyl sugar modified ribonucleotides; wherein the ribonucleotides at the 5′ terminus and the 3′ terminus of the antisense strand are 2′-O-methyl sugar modified; and wherein the ribonucleotides at the 5′ terminus and the 3′ terminus of the sense strand are unmodified. 
       
     
     
         57 . A double-stranded compound having the following structure:
   5′(N) x −Z3′(antisense strand)
     3′Z′−(N′) y 5′(sense strand)
   wherein each N and N′ is a ribonucleotide selected from the group consisting of a modified ribonucleotide or an unmodified ribonucleotide and each of (N) x  and (N′) y  is an oligomer in which each consecutive N or N′ is joined to the next N or N′ by a covalent bond;   wherein each of x and y is an integer between 18 and 40;   wherein each of Z and Z′ may be present or absent, but if present is dTdT and is covalently attached at the 3′ terminus of the strand in which it is present;   and wherein the sequence of (N) x  and the sequence of (N′) y  comprise an antisense sequence and corresponding sense sequence selected from Table A, set forth in any one of SEQ ID NOS: 3-1852.

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