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US 9,803,195 B2Granted
Natural antisense and non-coding RNA transcripts as drug targets
AssigneeThe Scripps Research Institute
InventorClaes Wahlestedt
Life of this patent
Estimating the term…
Abstract
Small interfering RNA (siRNA) knock down antisense transcripts, and regulate the expression of their sense partners. This regulation can either be discordant (antisense knockdown results in sense transcript elevation) or concordant (antisense knockdown results in concomitant sense transcript reduction).
Claims12
A method of treating Alzheimer's Disease comprising administering to a patient in need thereof at least one nucleic acid molecule comprising a sequence selected from:
- SEQ ID NOS:
- 4, 5, 6, 7, 8, 40, 41, 42, 43, 44, 45, 58, 59, 60, 61, 64, 65, 66, and 67.
A method of treating a disease or condition in a subject in need of treatment thereof by modulating expression of a target gene, the method comprising:
- targeting a nucleic acid molecule of 10 to 30 nucleotides in length to a natural anti-sense transcript of the target gene, wherein the target gene encodes CD97, TS-α, C/EBP delta, CDC23, PINK1, HIF1α, Gnbp3g, Adrenomedullin AM1 receptor, 6330439J10 (3-oxoacid CoA transferase), CtpW85 (Cathepsin W), Ddx-39, rTS-α, I530027A02, Kif20a, PINK-AS, aHIF1α, Gnbp3g-AS, AdmR-AS, A230019L24, BACE or CtpW-AS, wherein the nucleic acid molecule targeting the natural anti-sense transcript is complementary to the natural antisense transcript;
- and, binding of the nucleic acid molecule to the natural antisense transcript, whereby expression of the target gene is modulated.
Dependencies are inferred from the claim text.