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1.发明公开METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS 审中-公开有效EXON（44）跳过DUCHENNE肌肉萎缩症及相关手段的方法
公开(公告)号：EP3284823A2
公开(公告)日：2018-02-21
申请号：EP17195536.2
申请日：2009-05-14
发明人： Platenburg, Gerard Johannes , de Kimpe, Josephus Johannes , van Deutekom, Judith Christina Theodora , van Ommen, Garrit-Jan Boudewijn , Aartsma-Rus, Annemieke
IPC分类号： C12N15/11 , A61K31/7088 , C12N15/86
CPC分类号： C12N15/113 , A61K48/00 , C12N2310/11 , C12N2310/111 , C12N2310/314 , C12N2310/315 , C12N2310/321 , C12N2310/3233 , C12N2310/346 , C12N2310/3513 , C12N2310/3517 , C12N2320/33 , C12N2310/3521
摘要： The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5'-GUGGCUAACAGAAGCU (SEQ ID NO 1) and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.
摘要翻译： 本发明涉及与具有序列5'-GUGGCUAACAGAAGCU（SEQ ID NO 1）的核苷酸分子结合和/或互补的核酸分子，并涉及其在诱导DMD基因的外显子44跳跃 DMD患者。
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公开(公告)号：EP2636742B1
公开(公告)日：2017-04-19
申请号：EP13170251.6
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/113 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
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公开(公告)号：EP2636740A1
公开(公告)日：2013-09-11
申请号：EP13170239.1
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/113 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
摘要： The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
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公开(公告)号：EP2594642A1
公开(公告)日：2013-05-22
申请号：EP12198497.5
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/11 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
摘要： The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
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公开(公告)号：EP3400948A1
公开(公告)日：2018-11-14
申请号：EP18180952.6
申请日：2009-03-11
发明人： de Kimpe, Josephus Johannes , Platenburg, Gerardus Johannes , van Deutekom, Judith Christina Theodora , Aartsma-Rus, Annemieke , van Ommen, Garrit-Jan Boudewijn
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3521
摘要： The invention relates a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, exons 50-53 of the DMD pre-mRNA in a patient, preferably in an isolated cell of a patient, the method comprising providing said cell and/or said patient with a molecule. The invention also relates to said molecule as such.
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公开(公告)号：EP3382021A1
公开(公告)日：2018-10-03
申请号：EP18173900.4
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/11 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
摘要： The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
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公开(公告)号：EP2636740B1
公开(公告)日：2016-11-09
申请号：EP13170239.1
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/113 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
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公开(公告)号：EP2636741B1
公开(公告)日：2016-04-27
申请号：EP13170245.8
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/113 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
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9.发明授权Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA 有权的方法和手段进行有效SKIPPING外显子45 Duchenne型肌营养不良症，前体mRNA
公开(公告)号：EP2607484B1
公开(公告)日：2016-01-06
申请号：EP13160338.3
申请日：2009-01-13
发明人： de Kimpe, Josephus Johannes , Platenburg, Gerardus Johannes , van Deutekom, Judith Christina Theodora , Aartsma-Rus, Annemieke , van Ommen, Garrit-Jan Boudewijn
IPC分类号： C12N15/113 , A61K31/7105
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3521
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公开(公告)号：EP2594642B1
公开(公告)日：2015-12-30
申请号：EP12198497.5
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/11 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
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