发明公开
EP2850188A1 COMPOSITIONS AND METHODS FOR MODULATING HEMOGLOBIN GENE FAMILY EXPRESSION
审中-公开
组合物和方法调制表达血红蛋白基因家族的
- 专利标题： COMPOSITIONS AND METHODS FOR MODULATING HEMOGLOBIN GENE FAMILY EXPRESSION
- 专利标题（中）： 组合物和方法调制表达血红蛋白基因家族的
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申请号： EP13790998.2申请日： 2013-05-16
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公开(公告)号： EP2850188A1公开(公告)日： 2015-03-25
- 发明人: KRIEG, Arthur, M. , SUBRAMANIAN, Romesh , MCSWIGGEN, James , LEE, Jeannie, T.
- 申请人： Rana Therapeutics Inc. , The General Hospital Corporation d/b/a Massachusetts General Hospital
- 申请人地址： 790 Memorial Drive, Suite 203 Cambridge, MA 02139 US
- 专利权人： Rana Therapeutics Inc.,The General Hospital Corporation d/b/a Massachusetts General Hospital
- 当前专利权人： Rana Therapeutics Inc.,The General Hospital Corporation d/b/a Massachusetts General Hospital
- 当前专利权人地址： 790 Memorial Drive, Suite 203 Cambridge, MA 02139 US
- 代理机构： Hoffmann Eitle
- 优先权： US201261647901P 20120516; US201361785956P 20130314
- 国际公布： WO2013173599 20131121
- 主分类号： C12N15/11
- IPC分类号： C12N15/11 ; C07H21/00 ; A61K31/7125 ; A61P7/06
摘要：
Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes (HBB, HBD, HBE1, HBG1 or HBG2). Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes. Methods for modulating expression of hemoglobin genes using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of hemoglobin genes.
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