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1.发明公开Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA 有权VERFAHREN UND MITTEL ZUM WIRKSAMENÜBERSPRINGENVON EXON 45在DUCHENNE-MUSKELDYSTROPHIE-PRÄ-MRNA
公开(公告)号：EP2607484A1
公开(公告)日：2013-06-26
申请号：EP13160338.3
申请日：2009-01-13
申请人： Prosensa Technologies B.V. , Prosensa B.V. , ProSensa Holding BV , Academisch Ziekenhuis Leiden
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
摘要： The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon.
摘要翻译： 本发明涉及一种诱导或促进在Duchenne肌营养不良患者（优选在分离的（肌肉））细胞中跳脱DMD前mRNA的外显子45的方法，该方法包括向所述细胞提供结合连续伸展的分子 在所述外显子内至少21个核苷酸。
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公开(公告)号：EP2614827A2
公开(公告)日：2013-07-17
申请号：EP13160310.2
申请日：2008-10-27
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 , A61K31/56 , A61K31/57 , A61K31/573 , A61K31/58 , A61K31/7088 , A61K38/1719 , A61K45/06 , A61K48/00 , C12N2310/11 , C12N2310/111 , C12N2310/31 , C12N2310/313 , C12N2310/314 , C12N2310/315 , C12N2310/3181 , C12N2310/321 , C12N2310/3231 , C12N2310/3233 , C12N2310/346 , C12N2320/31 , C12N2320/33 , A61K2300/00
摘要： The invention provides means and methods for alleviating one or more symptom(s) of Duchenne Muscular Dystrophy and/or Becker Muscular Dystrophy. Therapies using compounds for providing patients with functional muscle proteins are combined with at least one adjunct compound for reducing inflammation, preferably for reducing muscle tissue inflammation, and/or at least one adjunct compound for improving muscle fiber function, integrity and/or survival.
摘要翻译： 本发明提供了用于缓解杜兴氏肌营养不良症和/或贝克肌营养不良症的一种或多种症状的手段和方法。 使用化合物为患者提供功能性肌肉蛋白质的疗法与至少一种用于减少炎症的辅助化合物（优选用于减少肌肉组织炎症）和/或用于改善肌肉纤维功能，完整性和/或存活的至少一种辅助化合物组合。
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3.发明公开Means and methods for counteracting muscle disorders 有权Mittel und Verfahren zurBekämpfungvon Muskelleiden
公开(公告)号：EP2614827A3
公开(公告)日：2013-09-25
申请号：EP13160310.2
申请日：2008-10-27
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 , A61K31/56 , A61K31/57 , A61K31/573 , A61K31/58 , A61K31/7088 , A61K38/1719 , A61K45/06 , A61K48/00 , C12N2310/11 , C12N2310/111 , C12N2310/31 , C12N2310/313 , C12N2310/314 , C12N2310/315 , C12N2310/3181 , C12N2310/321 , C12N2310/3231 , C12N2310/3233 , C12N2310/346 , C12N2320/31 , C12N2320/33 , A61K2300/00
摘要： The invention provides means and methods for alleviating one or more symptom(s) of Duchenne Muscular Dystrophy and/or Becker Muscular Dystrophy. Therapies using compounds for providing patients with functional muscle proteins are combined with at least one adjunct compound for reducing inflammation, preferably for reducing muscle tissue inflammation, and/or at least one adjunct compound for improving muscle fiber function, integrity and/or survival.
摘要翻译： 本发明提供了减轻Duchenne肌营养不良症和/或Becker肌营养不良症的一种或多种症状的方法和方法。 使用化合物为患者提供功能性肌肉蛋白的治疗与至少一种辅助化合物组合，用于减轻炎症，优选减少肌肉组织炎症，和/或至少一种辅助化合物，用于改善肌肉纤维功能，完整性和/或存活。
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4.发明公开Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means 有权Verfahrenfüreffizientes Exon（44）-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel
公开(公告)号：EP2813570A1
公开(公告)日：2014-12-17
申请号：EP14179979.1
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，其用于诱导在DMD基因的外显子44的跳跃的方法中的用途 DMD患者。
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5.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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