Oligonucleotides for inducing paternal ube3a expression
AssigneesRoche Innovation Center Copenhagen A/S, Hoffmann-La Roche Inc.
InventorsVeronica Costa, Maj HEDTJÄRN, Marius Hoener, Ravi Jagasia, Mads Aaboe Jensen, Christoph Patsch, Lykke Pedersen, Søren Vestergaard Rasmussen
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Abstract
The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.
Claims26
An antisense oligonucleotide which comprises a contiguous nucleotide sequence of 10 to 30 nucleotides in length, wherein the oligonucleotide comprises a sequence selected from the group consisting of SEQ ID NO:
- 46, 47, 48, 221, 222, 223, 224, 225, 289, 290, 291, 292, 293, 294, 295, 296, 297, 298, 299, 300, 301, 302, 303, 304, 304, 305, 585, 586, 587, 588, 589, 625, 627, 628, 629, 630, 631 632, 698, 699, 700, 702, 703, 704, 705, 706, 707, 708, 709, 710, 711, 712, 713, 714, 715, 716, 717, 718, 733, 734, 735, 736, 737, 738, 739, 740, 741, 742, 743, 744, 745, 746, 747, 748, 749, 750, 751, 752, 754, 755, 756, 757, 758, 759, 760, 761, 762, 763, 764, 765, 766, 767, 768, 769, 770, 772, 773, 774, 775, 776, 777, 778, 779, 780, 781, 782, 783, 784, 785, 786, 787, 788, 789, 790, 791, 792, 793, 794, 795, 796, 797, 798, 799, 800, 801, 802, 803, 804, 805, 806, 807.
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Dependencies are inferred from the claim text.