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AssigneePhyzat Biopharmaceuticals, Lda.
InventorPatrício Manuel VIEIRA ARAUJO SOARES DA SILVA
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The present disclosure relates to method of producing and using short interfering nucleic acids (siNAs) for preventing or reversing progressive optical neuropathy associated with the elevation of intraocular pressure due to excessive noradrenergic activation. In particular, this disclosure relates to the method of producing and using siNAs for or reversing progressive optical neuropathy, wherein the optical neuropathy is selected from the following list: diabetic retinopathy, infections, inflammation, uveitis and glaucoma, such as open-angle glaucoma, close-angle glaucoma, normal pressure glaucoma, congenital glaucoma, secondary glaucoma, pigmentary glaucoma, pseudoexfoliative glaucoma, traumatic glaucoma, neovascular glaucoma, endothelial iridocorneal syndrome and uveitic glaucoma. The present disclosure is also directed to interfering RNA duplexes and vectors encoding such interfering RNA duplexes.
An isolated or synthetic short interfering nucleic acid—(siNA)—molecule, wherein said molecule comprises a nucleic acid sequence selected from the group consisting of SEQ ID No 144, SEQ ID No 174, SEQ. ID No 233, SEQ ID No 235, SEQ ID No 239, SEQ ID No 250, SEQ ID No 265, SEQ ID No 269, SEQ ID No 281, SEQ ID No 311, SEQ ID No 370, SEQ ID No 372, SEQ ID No 376, SEQ ID No 387, SEQ ID No 402, SEQ ID No 406, and sequences comprising at least 18 contiguous nucleotides differing by no more than 4 nucleotides from the nucleotide sequence, and wherein said siNA molecule reduces expression of the dopamine-beta-hydroxylase—(DBH)—gene in a cell.
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A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of DBH-gene in a cell, wherein the dsRNA agent comprises a sense strand and an antisense strand forming a double stranded region, wherein the sense strand comprises a nucleic acid sequence selected from the group consisting of SEQ ID No 144, SEQ ID No 174, SEQ ID No 233, SEQ ID No 235, SEQ ID No 239, SEQ ID No 250, SEQ ID No 265, SEQ ID No 269, and sequences comprising at least 18 contiguous nucleotides differing by no more than 4 nucleotides from the nucleotide sequence, and wherein the antisense strand comprises a nucleic acid sequence selected from the group consisting of SEQ ID No 281, SEQ. ID No 311, SEQ ID No 370, SEQ ID No 372, SEQ ID No 376, SEQ ID No 387, SEQ ID No 402, SEQ ID No 406, and sequences comprising at least 18 contiguous nucleotides differing by no metre than 4 nucleotides from the nucleotide sequence.
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Dependencies are inferred from the claim text.