NATURAL ANTISENSE AND NON-CODING RNA TRANSCRIPTS AS DRUG TARGETS
First Claim
Patent Images
1. A pharmaceutical composition for treating neurological disorders, comprising at least one nucleic acid molecule and variants thereof of SEQ ID NOS:
- 1-67 in a pharmaceutically acceptable carrier.
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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).
242 Citations
37 Claims
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1. A pharmaceutical composition for treating neurological disorders, comprising at least one nucleic acid molecule and variants thereof of SEQ ID NOS:
- 1-67 in a pharmaceutically acceptable carrier.
- View Dependent Claims (2, 5, 6)
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3-4. -4. (canceled)
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7. (canceled)
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8. An expression vector comprising any one or more of SEQ ID NOS:
- 1-67 and variants thereof.
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9-10. -10. (canceled)
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11. A method of treating a neurological disorder comprising administering to a patient in need thereof at least one of SEQ ID NOS:
- 1-67 and variants thereof.
- View Dependent Claims (12, 13, 14, 15, 16)
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17. A method of up-regulating gene expression comprising:
- targeting a nucleic acid molecule to an anti-sense transcript of a sense strand, wherein the nucleic acid molecule targeting the anti-sense transcript is complementary to the anti-sense strand; and
, binding of the nucleic acid to the anti-sense transcript;
wherein, expression of the sense strand is elevated and expression of the gene is up-regulated. - View Dependent Claims (18, 19, 20)
- targeting a nucleic acid molecule to an anti-sense transcript of a sense strand, wherein the nucleic acid molecule targeting the anti-sense transcript is complementary to the anti-sense strand; and
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21-31. -31. (canceled)
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32. A composition comprising nucleic acid sequences targeting at least one gene comprising CD97, TS-α
- , C/EBP delta, CDC23, PINK1, HIF1α
, Gnbp3g, Adrenomedullin AM1 receptor, 6330439J10 (3-oxoacid CoA transferase), CtpW85 (Cathepsin W), Ddx-39, rTS-o, 1530027 A02, Kif20a, PINK-AS, aHIF1α
, Gnbp3g-AS, AdmR-AS, A230019L24, BACE or Ctp W-AS.
- , C/EBP delta, CDC23, PINK1, HIF1α
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33. A method of treating Parkinson'"'"'s Disease comprising administering to a patient in need thereof at least one of SEQ ID NO:
- 1-3 and variants thereof.
- View Dependent Claims (34)
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35. A method of diagnosing a neurological disease in a subject comprising:
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(a) obtaining biological tissue from the subject; (b) determining the level of BACE1-AS transcripts in said tissue; and (c) diagnosing said patient as having said neurological disease if the level of BACE1-AS transcripts is higher than a predetermined value. - View Dependent Claims (36, 37)
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Specification