US2020188532A1PendingUtilityA1

Demethylation to Treat Eye Disease

Assignee: UNIV CALIFORNIAPriority: Jun 11, 2018Filed: Feb 26, 2020Published: Jun 18, 2020
Est. expiryJun 11, 2038(~11.9 yrs left)· nominal 20-yr term from priority
C12Y 203/01C12N 2750/14143C12N 15/90C12N 15/1137A61P 27/02A61K 48/0075A61K 48/005A61K 31/706A61K 9/0048C12N 2310/20A61K 48/0058A61K 9/20
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Claims

Abstract

Methods for treating age-related eye diseases or conditions are provided. Methods for treating an age-related eye disease or condition in a subject by administering one or more demethylation compounds or agents are provided.

Claims

exact text as granted — not AI-modified
1 . A method for increasing an amount of ELOVL2 enzyme in the eye of a subject, comprising administering an effective amount of a formulation comprising a nucleic acid encoding an ELOVL2 enzyme to the eye of a subject in need. 
     
     
         2 . The method of  claim 1 , wherein the nucleic acid comprises a recombinant viral vector. 
     
     
         3 . The method of  claim 2 , wherein the viral vector is selected from an adenoviral vector, adeno-associated virus vector, lentivirus vector, vaccinia virus vector and retroviral vector. 
     
     
         4 . The method of  claim 1 , wherein the formulation further comprises one or more excipients suitable for administration to a human subject. 
     
     
         5 . The method of  claim 1 , wherein the formulation is administered to the eye by an intravitreal, subretinal, subconjunctival, subtenon, or posterior juxtascleral route. 
     
     
         6 . The method of  claim 1 , wherein the administration of the nucleic acid encoding the ELOVL2 enzyme treats age-related macular degeneration (AMD), diabetic eye disease, glaucoma, low vision or dry eye in the subject. 
     
     
         7 . The method of  claim 6 , wherein the age-related eye disease is dry AMD. 
     
     
         8 - 12 . (canceled) 
     
     
         13 . The method of  claim 1 , wherein the nucleic acid comprises a promoter that expresses in retinal cells. 
     
     
         14 . The method of  claim 13 , wherein the retinal cells comprise cells of the retinal pigment epithelium (RPE) and/or photoreceptors. 
     
     
         15 . The method of  claim 13 , wherein the promoter that expresses in the RPE is a VMD2 promoter. 
     
     
         16 . A method of increasing the amount of ELOVL2 protein production in an eye of a subject, comprising intraocularly injecting into the eye of a subject in need a formulation comprising a nucleic acid encoding an ELOVL2 protein effective to increase the amount of EVOLV2 protein in the eye. 
     
     
         17 . The method of  claim 16 , wherein, the subject is a human subject. 
     
     
         18 . The method of  claim 17 , wherein increasing the amount of ELOVL2 protein in the eye treats age-related macular degeneration (AMD) in the human subject. 
     
     
         19 . The method of  claim 18 , wherein treating AMD in the subject comprises treating dry age-related macular degeneration in the human subject. 
     
     
         20 . The method of  claim 16 , wherein the intraocular injection is administered via an intravitreal, subretinal, subconjunctival, subtenon, or posterior juxtascleral route. 
     
     
         21 . The method of  claim 16 , wherein the nucleic acid comprises an adenoviral vector comprising a promoter capable of expressing the ELOVL2 gene in retinal cells of the subject. 
     
     
         22 . The method of  claim 21 , wherein the promoter is a VMD2 promoter. 
     
     
         23 . The method of  claim 16 , wherein the method further comprises a vitrectomy prior to injection of the formulation. 
     
     
         24 . The method of  claim 16 , wherein the method comprises one or more intraocular injections of nucleic acid vectors encoding the ELOVL2 protein in 0.1 ml aliquots. 
     
     
         25 . The method of  claim 16 , wherein prior to the intraocular injection the subject has been treated with prednisone. 
     
     
         26 . The method of  claim 16 , wherein the intraocular injection comprises an injection into the subretinal space of the eye using a subretinal cannula.

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