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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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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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5.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：EP2614827B1
公开(公告)日：2017-06-28
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.
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公开(公告)号：EP2505211B1
公开(公告)日：2020-04-08
申请号：EP12174179.7
申请日：2008-07-14
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公开(公告)号：EP3238737A1
公开(公告)日：2017-11-01
申请号：EP17171389.4
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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9.发明公开
公开(公告)号：EP2505211A1
公开(公告)日：2012-10-03
申请号：EP12174179.7
申请日：2008-07-14
发明人： Heemskerk, Johannes Antonius , van Deutekom, Judith Christina Theodora , Van Kuik-Romeijn, Petra , Platenburg, Gerardus Johannes
CPC分类号： A61K49/0056 , A61K47/62 , A61K49/0032 , A61K49/0047
摘要： The invention provides conjugates, comprising an organ, tissue or tumor cell homing molecule linked to a moiety. Such a moiety can be, for example, an oligonucleotide, small interfering RNA, gene, virus, protein, pharmaceutical or detectable agent. In addition the invention provides methods to diagnose or treat a pathology of the muscle or heart, by administrating to a subject having or suspected of having a pathology a molecule or conjugate that homes to, binds to and is taken up by the muscle cells or heart cells.
摘要翻译： 本发明提供了缀合物，其包括连接到一个部分的器官，组织或肿瘤细胞归巢分子。 这样的部分可以是，例如，在寡核苷酸，小干扰RNA，基因，病毒，蛋白质，药物或可检测的试剂。 此外，本发明提供的方法来诊断或治疗肌肉或心脏的病理，通过给药至患有或者怀疑患有的病状的分子或缀合物做舍的受试者，结合并是由肌细胞或心脏吸收 细胞。
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