# US 10,369,231 B2 · MiR-204 and miR-211 and uses thereof > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 10,369,231 B2 - Document ID: US10369231B2 - Status: Granted - Kind: B2 - Application number: 14/774,586 - Filed: 2014-03-11 - Published: 2016-01-28 - Granted: 2019-08-06 - Earliest claimed: 2013-03-11 - Assignees: Fondazione Telethon - Applicants: Fondazione Telethon - Inventors: Sandro Banfi; Enrico Maria Surace; Ivan Conte; Marianthi Karali; Elena Marrocco - Web page: https://patentgrep.com/patent/US10369231B2 ## CPC - A61K 48/005: {characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered} - A61K 31/7105: Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links - A61P 27/02: Ophthalmic agents - C12N 15/113: Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)} - C12N 15/86: Viral vectors - C12N 2310/141: MicroRNAs, miRNAs - C12N 2710/10032: Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent - C12N 2710/10043: viral genome or elements thereof as genetic vector - A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D) - A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS - C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00) ## Abstract The present invention relates to at least one agent capable of increasing the level of one or more miRNA in a cell or cells of a subject, said miRNA comprising the sequence UUCCCUU, for use in the treatment and/or prevention of a retinal dystrophy, in particular characterized by photoreceptor degeneration, relative pharmaceutical compositions, nucleic acids, vectors and host cells. ## Claims ### Claim 1 A method for treating and/or preventing an inherited retinal dystrophy disease or disorder in a subject, the method comprising administering to the subject a member selected from the group consisting of: a) an agent selected from the group consisting of an miRNA, an miRNA precursor, a mature miRNA, an miRNA mimetic or a mixture of miRNA mimetics, an RNA or DNA molecule encoding for said miRNA, for said miRNA precursor, for said mature miRNA, for said miRNA mimetic or mixture of miRNA mimetics, and combinations thereof, b) a pharmaceutical composition containing said agent of a) and pharmaceutically acceptable excipients and/or diluents, c) a recombinant expression vector comprising a coding sequence for the agent of a), under the control of a suitable promoter, d) a host cell transformed by the recombinant expression vector of c) comprising a coding sequence for the agent, and e) combinations of a) through d); wherein the agent of a) comprises the seed sequence UUCCCUU or encodes a nucleotide sequence comprising a seed sequence UUCCCUU; and wherein the inherited retinal dystrophy disease or disorder is retinitis pigmentosa, Leber congenital amaurosis, a cone-rod dystrophy, or a cone dystrophy. ### Claim 2 (depends on 1) The method according to claim 1, wherein said agent of a) comprises the mature sequence of miR-204 or the mature sequence of miR-211. ### Claim 3 (depends on 1) The method according to claim 1, wherein said agent is provided by a delivery vehicle. ### Claim 4 (depends on 1) The method according to claim 1, wherein the inherited retinal dystrophy disease or disorder is Retinitis Pigmentosa. ### Claim 5 (depends on 1) The method according to claim 1, wherein the suitable promoter is the Rhodopsin promoter sequence. ### Claim 6 (depends on 1) The method according to claim 1, wherein the recombinant expression vector of d) further comprises one or more sequences coding for a wild type version of a sequence responsible for the inherited retinal dystrophy, under the control of a suitable promoter. ### Claim 7 (depends on 6) The method according to claim 6, wherein the one or more wild-type version of the coding sequence responsible for the retinal dystrophy is selected from the group consisting of: SEQ ID NO: 23 through SEQ ID NO: 414. ### Claim 8 (depends on 1) The method according to claim 1, wherein the recombinant expression vector of d) is an adeno-associated virus (AAV) derivative. ### Claim 9 (depends on 1) The method according to claim 1, wherein the nucleic acid sequence or the recombinant expression vector or the host cell is administered as part of a pharmaceutical composition containing pharmaceutically acceptable excipients and/or diluents for use in the treatment and/or prevention of a retinal dystrophy. ### Claim 10 (depends on 6) The method according to claim 6, wherein the sequences coding for a wild type version of a sequence responsible for the inherited retinal dystrophy are inserted in a recombinant expression vector that is separate from the recombinant expression vector of d). ### Claim 11 The method according to 26, wherein said agent is delivered via intraocular administration to said subject. ### Claim 12 (depends on 3) The method according to claim 3, wherein the delivery vehicle is selected from the group consisting of viral vectors, microspheres, liposomes, colloidal gold particles, lipopolysaccharides, polypeptides, polysaccharides, and pegylated viral vehicles. ### Claim 13 (depends on 3) The method according to claim 3, wherein the delivery vehicle is an adeno-associated virus (AAV) derivative. ### Claim 14 (depends on 1) The method according to claim 1, wherein the agent of a) protects the retina from the inherited retinal dystrophy disease or disorder. ### Claim 15 (depends on 1) The method according to claim 1, wherein the agent of a) treats the retina for the inherited retinal dystrophy disease or disorder.