发明申请
US20150025231A1 COMPOSITIONS AND METHODS FOR MODULATION OF IKBKAP SPLICING
审中-公开
用于IKBKAP分离调制的组合物和方法
- 专利标题: COMPOSITIONS AND METHODS FOR MODULATION OF IKBKAP SPLICING
- 专利标题(中): 用于IKBKAP分离调制的组合物和方法
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申请号: US14371941申请日: 2013-01-11
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公开(公告)号: US20150025231A1公开(公告)日: 2015-01-22
- 发明人: C. Frank Bennett , Frank Rigo , Adrian R. Krainer , Rahul Sinha
- 申请人: Isis Pharmaceuticals, Inc. , Cold Spring Harbor Laboratory
- 申请人地址: US CA Cold Spring Harbor US CA Carlsbad
- 专利权人: COLD SPRING HARBOR LABORATORY,ISIS PHARMACEUTICALS, INC.
- 当前专利权人: COLD SPRING HARBOR LABORATORY,ISIS PHARMACEUTICALS, INC.
- 当前专利权人地址: US CA Cold Spring Harbor US CA Carlsbad
- 国际申请: PCT/US2013/021311 WO 20130111
- 主分类号: C12N15/113
- IPC分类号: C12N15/113
摘要:
The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.
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