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公开(公告)号：EP3389670A1
公开(公告)日：2018-10-24
申请号：EP16871732.0
申请日：2016-12-05
发明人： SPECTOR, David, L. , DIERMEIER, Sarah, Daniela , RIGO, Frank , BENNETT, C., Frank , FREIER, Susan, M. , ARUN, Gayatri , CHANG, Kung-chi
IPC分类号： A61K31/7088 , A61P35/00 , C12N15/11 , C12N15/113
CPC分类号： A61K31/713 , A61K45/06 , C12N15/113 , C12N2310/113 , C12N2310/315 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , A61K2300/00 , C12N2310/321 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of a MaTAR in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate breast cancer in an individual in need.
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公开(公告)号：EP2802674A1
公开(公告)日：2014-11-19
申请号：EP13735797.6
申请日：2013-01-11
IPC分类号： C12Q1/68
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533 , C12N2320/33
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 IΚΑΡ 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 IΚΑΡ protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.
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3.发明公开COMPOUNDS AND METHODS FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS 审中-公开化合物和方法调制之间的蛋白质与靶核酸的相互作用
公开(公告)号：EP2582397A2
公开(公告)日：2013-04-24
申请号：EP11796395.9
申请日：2011-06-15
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/3525 , C12N2310/3533 , C12N2320/33
摘要： Provided herein are antisense compounds and methods for recruiting one or more non-cleaving protein to a target nucleic acid in a cell. In certain instances such recruitment of a non-cleaving protein alters the function or activity of the target nucleic acid. In certain such instances, the target nucleic acid a pre-mRNA and the recruitment of the non-cleaving protein results in a change in splicing of the pre-mRNA.
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4.发明公开
公开(公告)号：EP2442816A1
公开(公告)日：2012-04-25
申请号：EP10790221.5
申请日：2010-06-17
发明人： BENNETT, C., Frank , HUNG, Gene , RIGO, Frank , KRAINER, Adrian, R. , HUA, Yimin , PASSINI, Marco, A. , SHIHABUDDIN, Lamya , CHENG, Seng, H. , KLINGER, Katherine, W.
IPC分类号： A61K31/7088
CPC分类号： A61K31/7088 , A61K31/712 , C12N15/113 , C12N2310/11 , C12N2320/33
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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5.发明公开
公开(公告)号：EP3995580A2
公开(公告)日：2022-05-11
申请号：EP21187583.6
申请日：2014-08-11
IPC分类号： C12N15/113 , A61K48/00
摘要： Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
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公开(公告)号：EP2971142B1
公开(公告)日：2020-06-24
申请号：EP14767904.7
申请日：2014-03-14
发明人： RIGO, Frank , BENNETT, C., Frank
IPC分类号： C12N15/113
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公开(公告)号：EP3208347B1
公开(公告)日：2019-08-14
申请号：EP16203872.3
申请日：2011-02-08
发明人： BENNETT, C., Frank , HAYDEN, Michael , FREIER, Susan, M. , GREENLEE, Sarah , CARROLL, Jeffrey , WARBY, Simon , SWAYZE, Eric, E.
IPC分类号： C12Q1/68 , C12N15/11 , C07H21/04 , C12Q1/6811
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8.发明授权METHODS FOR MODULATING METASTASIS-ASSOCIATED-IN-LUNG-ADENOCARCINOMA-TRANSCRIPT-1(MALAT-1) EXPRESSION 有权调节与肺转移瘤相关的肺 - 腺癌 - 转录物-1（MALAT-1）表达的方法
公开(公告)号：EP2794880B1
公开(公告)日：2018-05-02
申请号：EP12860563.1
申请日：2012-12-21
发明人： BENNETT, C., Frank , FREIER, Susan, M. , MARCUSSON, Eric, G. , HSU, Ssucheng, J. , MACLEOD, Robert, A.
CPC分类号： C12N15/1136 , A61K31/711 , C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2320/30 , C12N2310/321 , C12N2310/3525
摘要： The present embodiments provide compounds and methods for reducing expression of Metastasis-Associated-in-Lung-Adenocarcinoma-Transcript-1 (MALAT-1) RNA and/or protein in an animal. Such methods are useful for treating cancer, such as colon cancer, intestinal cancer, lung cancer (e.g. non-small cell lung cancer), liver cancer, and/or prostate cancer. In various aspects, the cancer is a primary cancer.
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公开(公告)号：EP2475675B1
公开(公告)日：2016-11-16
申请号：EP10816206.6
申请日：2010-09-10
发明人： HUNG, Gene , BENNETT, C., Frank , FREIER, Susan, M. , KORDASIEWICZ, Holly , STANEK, Lisa , CLEVELAND, Don, W. , CHENG, Seng, H. , SHIHABUDDIN, Lamya
IPC分类号： C12N15/113 , A61K31/7125 , A61K31/712
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/345 , C12N2310/346 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
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