Inhibitors of RTP801 and their use in disease treatment

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 RTP801 gene and/or protein.

1 .- 12 . (canceled) 
     
     
         13 . A method of treating a patient suffering from a condition selected from a respiratory disorder, an eye disease, a microvascular disorder, a hearing disorder, a pressure sore or a spinal cord injury or disease comprising administering to the patient a pharmaceutical composition comprising a therapeutically effective amount of a double-stranded siRNA compound, which compound comprises an antisense strand and a sense strand wherein the sequence of ribonucleotides in the antisense strand comprises SEQ ID NO:38 or SEQ ID NO:44 so as to thereby treat the patient. 
     
     
         14 . A method according to  claim 13  wherein the condition is an eye disease selected from the group consisting of wet age-related macular degeneration (AMD), dry AMD, glaucoma, diabetic macular edema, and diabetic retinopathy. 
     
     
         15 . A method according to  claim 13 , wherein the condition is a hearing disorder selected from the group consisting of acoustic trauma, presbycusis, and cisplatin-induced deafness. 
     
     
         16 .- 35 . (canceled) 
     
     
         36 . The method according to  claim 14 , wherein the eye disease is dry AMD or wet AMD. 
     
     
         37 . The method according to  claim 13 , wherein the condition is a respiratory disorder. 
     
     
         38 . The method according to  claim 37 , wherein the respiratory disorder is COPD. 
     
     
         39 . The method according to  claim 38 , wherein the respiratory disorder is asthma. 
     
     
         40 . The method according to  claim 37 , wherein the respiratory disorder is chronic bronchitis. 
     
     
         41 . The method according to  claim 37 , wherein the respiratory disorder is emphysema. 
     
     
         42 . The method according to  claim 13 , wherein the condition is a microvascular disorder. 
     
     
         43 . (canceled) 
     
     
         44 . The method according to  claim 42 , wherein the microvascular disorder is acute renal failure. 
     
     
         45 - 55 . (canceled) 
     
     
         56 . A method of treating a patient suffering from a condition selected from the group consisting of a microvascular disorder, hearing loss, acute renal failure (ARF), glaucoma, acute respiratory distress syndrome (ARDS), acute lung and respiratory injuries, organ transplantation rejection, ischemic or ischemia-reperfusion injuries or conditions, ischemia-reperfusion injury following lung transplantation, organ transplantation including lung, liver, heart, pancreas, and kidney transplantation, nephrotoxicity, neurotoxicity, spinal cord injury, pressure sores, age-related macular degeneration (AMD), dry eye syndrome, oral mucositis and chronic obstructive pulmonary disease (COPD) comprising administering to the patient a pharmaceutical composition comprising a therapeutically effective amount of an siRNA compound which compound comprises an antisense strand and a sense strand, wherein the sequence of ribonucleotides in the antisense strand comprises SEQ ID NO:38 or SEQ ID NO:44 so as to thereby treat the patient. 
     
     
         57 . The method of  claim 13 , wherein the sequence of the antisense strand comprises SEQ ID NO:38. 
     
     
         58 . The method of  claim 13 , wherein the sequence of the antisense strand comprises SEQ ID NO:44. 
     
     
         59 . The method of  claim 56 , where the sequence of the antisense strand comprises SEQ ID NO:38. 
     
     
         60 . The method of  claim 56 , wherein the sequence of the antisense strand comprises SEQ ID NO:44.