Rtp801l sirna compounds and methods of use thereof

Abstract

The invention provides chemically modified siRNA oligonucleotides that target RTP801L, compositions comprising same and to the use of such molecules to treat, inter alia, respiratory diseases including acute and chronic pulmonary disorders, eye diseases including glaucoma and ION, microvascular disorders, angiogenesis- and apoptosis-related conditions, and hearing impairments.

1 - 28 . (canceled) 
     
     
         29 . A method for providing neuroprotection in a patient in need thereof, comprising administering to the patient a therapeutically effective dose of a RTP801L inhibitor, thereby providing neuroprotection in the patient. 
     
     
         30 . A method for treating a neurodegenerative disease in a patient in need thereof, comprising administering to the patient a therapeutically effective dose of a RTP801L inhibitor, thereby treating the neurodegenerative disease in the patient. 
     
     
         31 . The method of  claim 29 , wherein the neuroprotection comprises neuroprotection of the retina. 
     
     
         32 . The method of  claim 30 , wherein the neurodegenerative disease comprises neurodegeneration of the retina and is associated with neuroretinal apoptosis. 
     
     
         33 . The method of  claim 32 , wherein the neurodegeneration is associated with diseases of the optic nerve. 
     
     
         34 . The method of  claim 31 , wherein the patient is suffering from glaucoma. 
     
     
         35 . The method of  claim 31 , wherein the patient is suffering from optic neuritis. 
     
     
         36 . The method of  claim 31 , wherein the patient is suffering from ischemic optic neuropathy (ION). 
     
     
         37 . The method of  claim 36 , wherein the ION is anterior ischemic optic neuropathy (AION). 
     
     
         38 . The method of  claim 30 , wherein the patient is suffering from retinopathy. 
     
     
         39 . The method of  claim 38 , wherein the retinopathy comprises retinal vascular damage or occlusion, retinopathy associated with systemic diseases, retinopathy associated with neurodegenerative diseases in the CNS, hypertensive retinopathy, radiation retinopathy, idiopathic retinal vasculitis, aneurysms, neuroretinitis, Birdshot retinochoroidopathy, long-standing retinal detachment acute glaucoma, wet age-related macular degeneration, dry age-related macular degeneration, geographic atrophy or retinitis pigmentosa. 
     
     
         40 . The method of  claim 39 , wherein the retinal vascular damage or occlusion comprises retinal artery occlusion, retinal vein occlusion, branch retinal artery or vein occlusion, retinopathy of prematurity, retinal embolization or Purtscher retinopathy. 
     
     
         41 . The method of  claim 38 , wherein the retinopathy is associated with a systemic disease selected from HIV retinopathy, retinopathy associated with systemic vasculitis, retinopathy associated with autoimmune diseases, or retinopathy associated with systemic neurodegenerative diseases. 
     
     
         42 . The method of  claim 29 , wherein the neuroprotection comprises neuroprotection of the inner ear. 
     
     
         43 . The method of  claim 29 , wherein the neuroprotection comprises neuroprotection of the CNS. 
     
     
         44 . A method for treating or preventing a disease associated with ischemia or ischemia-reperfusion injury to a tissue in a patient in need thereof, comprising administering to the patient a therapeutically effective dose of a RTP801L inhibitor, thereby treating or preventing the disease in the patient. 
     
     
         45 . The method of  claim 44 , wherein the tissue comprises skin, lung, heart or kidney. 
     
     
         46 . The method of  claim 29 , wherein the RTP801L inhibitor comprises an oligonucleotide. 
     
     
         47 . The method of  claim 46 , wherein the oligonucleotide is selected from an antisense oligonucleotide, a siRNA, an aptamer, or a catalytic RNA. 
     
     
         48 . A method for treating a disease associated with cell loss in a patient in need thereof, comprising administering to the patient a therapeutically effective dose of a RTP801L inhibitor, thereby treating the disease in the patient.