IP Library › Patent Application 16141781
Patent Application
App. No. 16/141,781

SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF

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Quick Facts
Patent No.
US None
App. No.
16/141,781
Abstract

The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount, activity, or expression of the target nucleic acid in a cell. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.

Claims (23)

1 .- 40 . (canceled)

41 . A compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the linked nucleosides comprise at least 8 contiguous nucleobases of a nucleobase sequence recited in SEQ ID NO: 5, 6, 27, 28, 29, 30, 38, 39, or 46.

42 . The compound of claim 41 , wherein the nucleobase sequence of the modified oligonucleotide is 100% complementary to SEQ ID NO. 1.

43 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 5.

44 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 6.

45 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 27.

46 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 28.

47 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 29.

48 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 30.

49 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 38.

50 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 39.

51 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 46.

52 . The compound of claim 43 , wherein the oligonucleotide has a ekek-d9-keke sugar motif.

53 . The compound of claim 44 , wherein the oligonucleotide has a kekk-d8-keke sugar motif.

54 . The compound of claim 45 , wherein the oligonucleotide has a ekkk-d8-kke sugar motif.

55 . The compound of claim 46 , wherein the oligonucleotide has a ekk-d8-kkke sugar motif.

56 . The compound of claim 47 , wherein the oligonucleotide has a ekekk-d8-keke sugar motif.

57 . The compound of claim 48 , wherein the oligonucleotide has a ekek-d8-kkeke sugar motif.

58 . The compound of claim 49 , wherein the oligonucleotide has a eekk-d8-kkeee sugar motif.

59 . The compound of claim 50 , wherein the oligonucleotide has a eekk-d8-kkeee sugar motif.

60 . The compound of claim 51 , wherein the oligonucleotide has a eeekk-d8-kkee sugar motif.

61 . The compound of claim 41 , comprising at least one phosphorothioate internucleoside linkage.

62 . A method of ameliorating a symptom of Huntington's disease, comprising administering an oligomeric compound of claim 41 to an animal in need thereof.