发明申请
US20150252364A1 COMPOSITIONS AND METHODS FOR MODULATING SMN GENE FAMILY EXPRESSION
审中-公开
用于调节SMN基因家族表达的组合物和方法
- 专利标题： COMPOSITIONS AND METHODS FOR MODULATING SMN GENE FAMILY EXPRESSION
- 专利标题（中）： 用于调节SMN基因家族表达的组合物和方法
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申请号： US14401194申请日： 2013-05-16
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公开(公告)号： US20150252364A1公开(公告)日： 2015-09-10
- 发明人: Arthur M. Krieg , Romesh Subramanian , James McSwiggen , Jeannie T. Lee
- 申请人： RaNA Therapeutics, Inc. , The General Hospital Corporation d/b/a Massachusetts General Hospital
- 申请人地址： US MA Cambridge US MA Boston
- 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： US MA Cambridge US MA Boston
- 国际申请： PCT/US2013/041440 WO 20130516
- 主分类号： C12N15/113
- IPC分类号： C12N15/113
摘要：
Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 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 SMN1 or SMN2.
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