[发明专利]用于诱导父本UBE3A表达的寡核苷酸在审
申请号: | 202211275978.X | 申请日: | 2016-11-11 |
公开(公告)号: | CN116064540A | 公开(公告)日: | 2023-05-05 |
发明(设计)人: | 韦罗妮卡·科斯塔;马伊·黑特耶恩;马里乌斯·赫纳;拉维·姚高希亚;马斯·奥波尔·詹森;克里斯托弗·帕奇;吕克·彼泽森;索伦·韦斯特高·拉斯穆森 | 申请(专利权)人: | 豪夫迈·罗氏有限公司 |
主分类号: | C12N15/113 | 分类号: | C12N15/113;C12N15/11;A61K31/7088;A61P25/00;A61P25/08;A61P25/14;A61P25/20;A61P25/28 |
代理公司: | 中科专利商标代理有限责任公司 11021 | 代理人: | 张莹;程金山 |
地址: | 瑞士*** | 国省代码: | 暂无信息 |
权利要求书: | 查看更多 | 说明书: | 查看更多 |
摘要: | |||
搜索关键词: | 用于 诱导 父本 ube3a 表达 寡核苷酸 | ||
1.反义寡核苷酸,所述反义寡核苷酸包含长度为10至30个核苷酸的连续核苷酸序列,所述连续核苷酸序列与人15号染色体上的位置25278410至25419462具有至少98%的互补性。
2.根据权利要求1所述的寡核苷酸,其中所述连续核苷酸序列与SEQ ID NO:1和/或2的靶核酸的区域互补。
3.根据权利要求1或权利要求2所述的寡核苷酸,其中所述连续核苷酸序列与SEQ IDNO:1所示的位置1-55318的靶核酸区域100%互补。
4.根据权利要求1-3任一项所述的寡核苷酸,所述连续核苷酸序列与靶核酸的子序列互补,其中所述子序列选自由表1或2所示的区域组成的组。
5.根据权利要求1-4任一项所述的寡核苷酸,其中所述寡核苷酸包含选自由以下组成的组的序列:SEQ ID NO:4,5,6,7,8,9,10,11,12,13,14,15,16,17,1819,20,21,22,23,23,24,25,26,26,27,28,29,30,31,31,32,33,34,35,36,37,38,39,40,41,42,43,44,44,45,45,46,47,48,49,50,51,52,53,53,54,54,55,56,57,58,59,60,61,62,63,64,64,65,66,67,68,69,70,71,72,73,74,75,76,77,78,79,79,80,81,8283,84,85,86,87,88,89,90,91,92,93,94,95,95,96,96,96,97,98,99,100,101,101,102,103,104,105,106,107,108,109,110,111,112,113,114,115,116,117,118,119,120,121,122,123,124,125,126,127,128,129,130,131,132,133,134,135,136,137,138,139,140,141,142,143,144,145,146,147,148,149,150,151,152,153,154,155,156,157,158,159,160,161,162,163,164,165,166,167,168,169,170,171,172,173,174,175,176,177,178,179,180,181,182,183,184,185,186,187,188,189,190,191,192,193,194,195,196,197,198,199,200,201,202,203,204,205,206,207,208,209,210,211,212,213,214,215,216,217,218,219,220,221,222,223,224,225,226,227,228,229,230,231,232,233,234,235,236,237,238,239,240,241,242,243,244,245,246,247,248,249,250,251,252,253,254,255,256,257,258,259,260,261,262,263,264,265,266,267,268,269,270,271,272,273,274,275,276,277,278,279,280,281,282,283,284,285,286,287,288,289,290,291,292,293,294,295,296,297,298,299,300,301,302,303,304,304,305,306,307,308,309,310,311,312,313,314,315,316,317,318,319,320,321,322,323,324,325,326,327,328,329,330,331,332,333,334,335,336,337,338,339,340,341,342,343,344,345,346,347,348,349,350,351,352,353,354,355,356,357,358,359,360,361,362,363,364,365,366,367,368,369,370,371,372,373,374,375,376,377,378,379,380,381,382,383,384,385,386,387,388,389,390,391,392,393,394,395,396,397,398,399,400,401,402,403,404,405,406,407,408,409,410,411,412,413,414,415,416,417,418,419,420,421,422,423,424,425,426,427,428,429,430,431,432,433,434,435,436,437,438,439,440,441,442,443,444,445,446,447,448,449,450,451,452,453,454,455,456,457,458,459,460,461,462,463,464,465,466,467,468,469,470,471,472,473,474,475,476,477,478,479,480,481,482,483,484,485,486,487,488,489,490,491,492,493,494,495,496,497,498,499,500,501,502,503,504,505,506,507,508,509,510,511,512,513,514,515,516,517,518,519,520,521,522,523,524,525,526,527,528,529,530,531,532,533,534,535,536,537,538,539,540,541,542,543,544,545,546,547,548,549,550,551,552,553,554,555,556,557,558,559,560,561,562,563,564,565,566,567,568,569,570,571,572,573,574,575,576,577,578,579,580,581,582,583,584,585,586,587,588,589,590,591,592,593,594,595,596,596,597,598,599,600,601,602,603,604,605,606,607,608,609,610,611,612,613,614,615,616,617,618,619,620,621,622,623,624,625,626,627,628,629,630,631,632,633,634,635,636,637,638,639,640,641,642,643,644,645,646,647,648,649,650,651,652,653,654,655,656,657,658,659,660,661,662,663,664,665,666,667,668,669,670,671,672,673,674,675,676,677,678,679,680,681,682,683,684,685,686,687,688,689,690,691,692,693,694,695,696,697,698,699,700,702,703,704,705,706,707,708,709,710,711,712,713,714,715,716,717,718,719,719,720,721,722,723,724,725,726,727,728,729,730,731,732,733,734,735,736,737,738,739,740,741,742,743,744,745,746,747,748,749,750,751,752,754,755,756,757,758,759,760,761,762,763,764,765,766,767,768,769,770,772,773,774,775,776,777,778,779,780,781,782,783,784,785,786,787,788,789,790,791,792,793,794,795,796,797,798,799,800,801,802,803,804,805,806,807,808,809,810,811,812,813,814,815,816,817和818。
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