Natural antisense and non-coding RNA transcripts as drug targets
AssigneeThe Scripps Research Institute
InventorClaes Wahlestedt
Life of this patent
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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).
Claims16
A pharmaceutical composition comprising at least one modified nucleic acid molecule targeting a gene encoding a drug target for a neurological disorder, wherein the at least one modified nucleic acid molecule binds to a naturally-occurring anti-sense transcript of a sense strand of the targeted gene encoding the drug target, wherein the at least one modified nucleic acid molecule is about 10 to about 22 residues in length, wherein the at least one modified nucleic acid molecule comprises at least one modified base or sugar moiety, wherein the at least one modified nucleic acid molecule that binds to the naturally-occurring anti-sense transcript is complementary to the naturally-occurring anti-sense transcript, and wherein the targeted gene is selected from the group consisting of PINK1, HIF1α, Gnbp3g, Adrenomedullin AM1 receptor, 3-oxoacid CoA transferase, Cathepsin W and BACE1.
A pharmaceutical composition comprising at least one modified nucleic acid molecule targeting a gene encoding a drug target for a neurological disorder, wherein the at least one modified nucleic acid molecule binds to a naturally-occurring anti-sense transcript of a sense strand of the targeted gene encoding the drug target, wherein the at least one modified nucleic acid molecule is about 10 to about 22 residues in length, wherein the at least one modified nucleic acid molecule comprises at least one modified base or sugar moiety, wherein the at least one modified nucleic acid molecule that binds to the naturally-occurring anti-sense transcript is complementary to the naturally-occurring anti-sense transcript, and wherein the neurological disorder is Alzheimer's Disease or Parkinson's Disease.
Dependencies are inferred from the claim text.