Item request has been placed! ×
Item request cannot be made. ×
loading  Processing Request

METHODS FOR SELECTIVE TARGETING OF HETEROCHROMATIN FORMING NON-CODING RNA

Item request has been placed! ×
Item request cannot be made. ×
loading   Processing Request
اقرأ أكثر حفظ في قائمتي
  • Publication Date:
    August 13, 2015
  • معلومة اضافية
    • Document Number:
      20150225722
    • Appl. No:
      14/699130
    • Application Filed:
      April 29, 2015
    • نبذة مختصرة :
      Provided herein are oligonucleotides that are useful for modulating the heterochromatin state of genes; related compositions and methods are also provided. In some embodiments, methods are provided for treating a disease associated with heterochromatin formation, including diseases associated with repeat expansion within genes.
    • Assignees:
      RaNA Therapeutics, Inc. (Cambridge, MA, US)
    • Claim:
      1.-53. (canceled)
    • Claim:
      54. A method for increasing expression of a target gene in cells, the method comprising: delivering to the cells an oligonucleotide complementary to a heterochromatin forming non-coding RNA associated with the target gene, wherein the oligonucleotide comprises a region of complementarity that is complementary with a position adjacent to a repeat region of the heterochromatin forming non-coding RNA.
    • Claim:
      55. The method of claim 54, wherein the oligonucleotide further comprises a region of complementarity that is complementary with a position within the repeat region of the heterochromatin forming non-coding RNA.
    • Claim:
      56. The method of claim 54, wherein the oligonucleotide is a cleavage promoting oligonucleotide.
    • Claim:
      57. The method of claim 54, wherein the RNA is a long non-coding RNA (lncRNA).
    • Claim:
      58. The method of claim 57, wherein the lncRNA is antisense to the gene.
    • Claim:
      59. The method of claim 54, wherein the repeat region comprises triplet repeats.
    • Claim:
      60. The method of claim 54, wherein the oligonucleotide comprises a region of complementarity that is complementary with a position within 5 kb from an end of the repeat region.
    • Claim:
      61. The method of claim 54, wherein the oligonucleotide is single stranded.
    • Claim:
      62. The method of claim 61, wherein the oligonucleotide comprises at least one modified nucleotide.
    • Claim:
      63. The method of claim 62, wherein the modified nucleotide is a bridged nucleotide.
    • Claim:
      64. The method of claim 63, wherein the bridge nucleotide is a locked nucleic acid (LNA) nucleotide, a constrained ethyl (cEt) nucleotide, or an ethylene bridged nucleic acid (ENA) nucleotide.
    • Claim:
      65. The method of claim 62, wherein the modified nucleotide is 2′ O-methyl nucleotide.
    • Claim:
      66. The method of claim 56, wherein the oligonucleotide is a gapmer.
    • Current International Class:
      12
    • الرقم المعرف:
      edspap.20150225722