MiR-204 and miR-211 and uses thereof
AssigneeFondazione Telethon
InventorsSandro Banfi, Enrico Maria Surace, Ivan Conte, Marianthi Karali, Elena Marrocco
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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.
Claims15
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.
The method according to 26, wherein said agent is delivered via intraocular administration to said subject.
Dependencies are inferred from the claim text.