US2002194630A1PendingUtilityA1

Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

43
Priority: Mar 15, 1999Filed: Mar 4, 2002Published: Dec 19, 2002
Est. expiryMar 15, 2019(expired)· nominal 20-yr term from priority
Inventors:William ManningVaravani DwarkiKatherine RendahlShangzhen ZhouLaura McgeeDana LauJohn G. FlannerySheldon S. MillerFei WangAdriana Di Polo
A01K 2267/03A61K 38/179A01K 67/0275A61K 38/57A61K 38/185C12N 2799/025C07K 14/50C07K 14/52A61K 48/00A01K 2217/052
43
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Claims

Abstract

Gene delivery vectors, such as, for example, recombinant adeno-associated viral vectors, and methods of using such vectors are provided for use in treating or preventing diseases of the eye.

Claims

exact text as granted — not AI-modified
We claim:  
     
         1 . A method of treating or preventing diseases of the eye, comprising, administering intraocularly a gene delivery vector which directs the expression of a neurotrophic factor, such that said disease of the eye is treated or prevented.  
     
     
         2 . The method according to  claim 1  wherein said neurotrophic factor is NGF, BDNF, CNTF, NT-3, or, NT-4.  
     
     
         3 . The method according to  claim 1  wherein said neurotrophic factor is a FGF.  
     
     
         4 . The method according to  claim 3  wherein said FGF is FGF-2, FGF-5, FGF-18, FGF-20, or, FGF-21.  
     
     
         5 . The method according to  claim 1  wherein said disease of the eye is macular degeneration.  
     
     
         6 . The method according to  claim 1  wherein said disease of the eye is diabetic retinopathy.  
     
     
         7 . The method according to  claim 1  wherein said disease of the eye is an inherited retinal degeneration.  
     
     
         8 . The method according to  claim 7  wherein said inherited retinal degeneration is retinitis pigmentosa.  
     
     
         9 . The method according to  claim 1  wherein said disease of the eye is glaucoma.  
     
     
         10 . The method according to  claim 1  wherein said disease of the eye is a surgery-induced retinopathy.  
     
     
         11 . The method according to  claim 1  wherein said disease of the eye is retinal detachment.  
     
     
         12 . The method according to  claim 1  wherein said disease of the eye is a photic retinopathy.  
     
     
         13 . The method according to  claim 1  wherein said disease of the eye is a toxic retinopathy.  
     
     
         14 . The method according to  claim 1  wherein said disease of the eye is a trauma-induced retinopathy.  
     
     
         15 . The method according to  claim 1  wherein said gene delivery vector is a retrovirus selected from the group consisting of HIV and FIV.  
     
     
         16 . The method according to  claim 1  wherein said gene delivery vector is a recombinant adeno-associated viral vector.  
     
     
         17 . A method of inhibiting neovascular disease of the eye, comprising, administering intraocularly a gene delivery vector which directs the expression of an anti-angiogenic factor, such that said neovascular disease of the eye is inhibited.  
     
     
         18 . The method according to  claim 17  wherein said anti-angiogenic factor is soluble Flt-1, PEDF, soluble Tie-2 receptor, or, a single chain anti-VEGF antibody.  
     
     
         19 . The method according to  claim 17  wherein said neovascular disease of the eye is diabetic retinopathy, wet AMD, and retinopathy of prematurity.  
     
     
         20 . The method according to  claim 17  wherein said gene delivery vector is a retrovirus selected from the group consisting of HIV and FIV.  
     
     
         21 . The method according to  claim 17  wherein said gene delivery vector is a recombinant adeno-associated viral vector.  
     
     
         22 . A gene delivery vector which directs the expression of a neurotrophic factor, or an anti-angiogenic factor.  
     
     
         23 . The gene delivery vector according to  claim 22  wherein said neurotrophic factor is NGF, BDNF, CNTF, NT-3, or, NT-4.  
     
     
         24 . The gene delivery vector according to  claim 22  wherein said neurotrophic factor is a FGF.  
     
     
         25 . The gene delivery vector according to  claim 22  wherein said FGF is FGF-2, FGF-5, FGF-18, FGF-20, or, FGF-21.  
     
     
         26 . The gene delivery vector according to  claim 22  wherein said anti-angiogenic factor is soluble Flt-1, PEDF, soluble Tie-2 receptor, or, a single chain anti-VEGF antibody.  
     
     
         27 . The gene delivery vector according to  claim 22  wherein said vector is generated from a retrovirus.  
     
     
         28 . The gene delivery vector according to  claim 27  wherein said retrovirus is HIV or FIV.  
     
     
         29 . The gene delivery vector according to  claim 22  wherein said vector is generated from a recombinant adeno-associated virus.  
     
     
         30 . A non-human animal model of neovascularization of the eye, comprising an animal having an angiogenic transgene in the eye.  
     
     
         31 . The non-human animal model according to  claim 30  wherein said neovascularization is retinal neovascularization.  
     
     
         32 . The non-human animal model according to  claim 30  wherein said neovascularization is choroidal neovascularization.  
     
     
         33 . The non-human animal model according to  claim 30  wherein said animal is a mouse or rat.  
     
     
         34 . The non-human animal model according to  claim 30  wherein said angiogenic transgene encodes VEGF.  
     
     
         35 . The non-human animal model according to  claim 30  wherein said angiogenic transgene encodes an angiopoietin.  
     
     
         36 . A method for making a non-human animal model of neovascularization of the eye, comprising administering to a non-human animal a gene delivery vector which directs the expression of an angiogenic transgene.  
     
     
         37 . The method according to  claim 36  wherein said gene delivery vector is administered subretinally.  
     
     
         38 . The method according to  claim 36  wherein said gene delivery vector is administered intravitreally.  
     
     
         39 . The method according to  claim 36  wherein said gene delivery vector is rAV or rAAV.  
     
     
         40 . The method according to  claim 36  wherein said angiogenic transgene is a nucleic acid molecule which encodes VEGF.  
     
     
         41 . The method according to  claim 36  wherein said angiogenic transgene is a nucleic acid molecule which encodes an angiopoietin.  
     
     
         42 . A method for determining the ability of an anti-angiogenic factor to inhibit neovascularization of the eye, comprising: (a) administering to an animal model according to any one of  claims 30  to  35  an anti-angiogenic factor, and (b) determining the ability of said anti-angiogenic factor to inhibit neovascularization of the eye.  
     
     
         43 . The method according to  claim 42  wherein said anti-angiogenic factor is administered subretinally.  
     
     
         44 . The method according to  claim 42  wherein said anti-angiogenic factor is administered intravitreally.

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