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公开(公告)号：US20110015384A1
公开(公告)日：2011-01-20
申请号：US12866608
申请日：2009-02-06
摘要： The use of a sulfurizing agent of formula A: or a salt, hydrate, solvate, or a mixture thereof, in which all R groups, independently, represent H or an organic group, in sulfurization. The sulfurizing agent is preferably a formamidine disulfide. It is found a particularly suitable alternative to existing sulfurizing agents, since it is easy to synthesize from readily available, cheap starting materials.
摘要翻译： 使用式A的硫化剂或其盐，水合物，溶剂合物或其混合物，其中所有R基团在硫化中独立地表示H或有机基团。 硫化剂优选为甲脒二硫化物。 发现现有的硫化剂是特别合适的替代方案，因为它容易从易得的廉价原料合成。
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2.发明授权公开(公告)号：US09926557B2
公开(公告)日：2018-03-27
申请号：US13094548
申请日：2011-04-26
申请人： Josephus Johannes De Kimpe , Gerardus Johannes Platenburg , Judith Christina Theodora Van Deutekom , Annemieke Aartsma-Rus , Garrit-Jan Boudewijn Van Ommen
发明人： Josephus Johannes De Kimpe , Gerardus Johannes Platenburg , Judith Christina Theodora Van Deutekom , Annemieke Aartsma-Rus , Garrit-Jan Boudewijn Van Ommen
IPC分类号： C12N15/113 , A61K31/56 , A61K31/57 , A61K38/17 , A61K31/7088 , A61K45/06 , A61K31/573 , A61K31/58 , A61K48/00
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 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 an isolated muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.
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3.发明申请METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50-53 审中-公开至少有效排除人类杜氏菌属基因的以下之一的方法和手段：43,46,50-53公开(公告)号：US20110263682A1
公开(公告)日：2011-10-27
申请号：US13094571
申请日：2011-04-26
申请人： Josephus Johannes De Kimpe , Gerardus Johannes Platenburg , Judith Christina Theodora Van Deutekom , Annemieke Aartsma-Rus , Garrit-Jan Boudewijn Van Ommen
发明人： Josephus Johannes De Kimpe , Gerardus Johannes Platenburg , Judith Christina Theodora Van Deutekom , Annemieke Aartsma-Rus , Garrit-Jan Boudewijn Van Ommen
IPC分类号： A61K31/7088 , C12N5/071 , C12N15/85 , C07H21/02 , A61P21/00
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 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.
摘要翻译： 本发明涉及一种方法，其中分子用于诱导和/或促进患者，优选在患者的分离的细胞中的DMD前体mRNA的外显子43，外显子46，外显子46-5，外显子50-53中的至少一个的跳跃 所述方法包括向所述细胞和/或所述患者提供分子。 本发明也涉及所述分子。
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4.发明授权Methods and means for treating DNA repeat instability associated genetic disorders 有权治疗DNA重复不稳定相关遗传疾病的方法和手段公开(公告)号：US08263760B2
公开(公告)日：2012-09-11
申请号：US12852057
申请日：2010-08-06
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/331 , C12N2310/346 , C12N2310/3521
摘要： The current invention provides for methods and medicaments that apply an oligonucleotide comprising aninosine and/or an uracile and/or a nucleotide containing a base able to form a wobble base pair, said oligonucleotide being preferably RNAse H substantially independent and being complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to a modified oligonucleotide which can be applied in a method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.
摘要翻译： 本发明提供了应用包含肌氨酸和/或尿嘧啶的寡核苷酸和/或含有能够形成摆动碱基对的碱基的核苷酸的方法和药物，所述寡核苷酸优选为RNA酶H，其基本上独立且仅与重复性 在人基因转录物中的序列，用于制备用于诊断，治疗或预防人类中与顺式元件重复不稳定相关的遗传疾病的药物。 因此，本发明提供了一种治疗与顺式元件重复不稳定相关的遗传疾病的方法。 本发明还涉及可以在本发明的方法中应用的修饰的寡核苷酸，以防止细胞中重复扩增的转录物的积累和/或翻译。
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公开(公告)号：US20120046342A1
公开(公告)日：2012-02-23
申请号：US13266110
申请日：2010-04-26
申请人： Judith Christina Theodora Van Deutekom , Josephus Johannes De Kimpe , Gerardus Johannes Platenburg
发明人： Judith Christina Theodora Van Deutekom , Josephus Johannes De Kimpe , Gerardus Johannes Platenburg
IPC分类号： A61K31/7088 , C07H21/04 , A61P29/00 , A61K31/7125 , A61P21/00 , C07H21/02 , A61K31/712
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/3181 , C12N2310/321 , C12N2310/3231 , C12N2310/331 , C12N2310/333 , C12N2310/336 , C12N2320/33 , C12N2310/3521
摘要： The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.
摘要翻译： 本发明提供了包含肌苷的寡核苷酸和/或含有能够形成摆动碱基对或其功能等同物的碱基的核苷酸，其中所述寡核苷酸或其功能等同物包含与至少部分互补的序列 肌营养不良蛋白前体RNA外显子或肌营养不良蛋白前体RNA的非外显子区域的至少一部分，所述部分是包含至少8个核苷酸的连续拉伸。 本发明还提供了所述寡核苷酸用于预防或治疗DMD或BMD的用途。
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6.发明申请METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-mRNA 有权在杜松子宫颈鳞状上皮细胞中有效移植外显子45的方法和手段公开(公告)号：US20120022134A1
公开(公告)日：2012-01-26
申请号：US13094548
申请日：2011-04-26
申请人： JOSEPHUS JOHANNES DE KIMPE , GERARDUS JOHANNES PLATENBURG , JUDITH CHRISTINA THEODORA VAN DEUTEKOM , ANNEMIEKE AARTSMA-RUS , GARRIT-JAN BOUDEWIJN VAN OMMEN
发明人： JOSEPHUS JOHANNES DE KIMPE , GERARDUS JOHANNES PLATENBURG , JUDITH CHRISTINA THEODORA VAN DEUTEKOM , ANNEMIEKE AARTSMA-RUS , GARRIT-JAN BOUDEWIJN VAN OMMEN
IPC分类号： A61K31/7088 , C12N15/63 , A61P21/00 , C12N15/113
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 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. The invention further relates to such molecule used in said method.
摘要翻译： 本发明涉及一种用于诱导或促进在Duchenne肌营养不良患者（优选在分离的（肌肉））细胞中跳跃DMD前体mRNA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 在所述外显子中至少21个核苷酸。 本发明还涉及用于所述方法的这种分子。
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7.发明申请METHODS AND MEANS FOR TREATING DNA REPEAT INSTABILITY ASSOCIATED GENETIC DISORDERS 有权用于治疗DNA重复不稳定相关遗传病的方法和手段公开(公告)号：US20110184050A1
公开(公告)日：2011-07-28
申请号：US12852057
申请日：2010-08-06
IPC分类号： A61K31/7088 , C07H21/00 , C07H21/02 , C07H21/04 , C12N15/63 , A61K48/00 , C12N5/071 , C12N15/86 , C12N15/85 , C12Q1/68 , A61P43/00
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/331 , C12N2310/346 , C12N2310/3521
摘要： The current invention provides for methods and medicaments that apply an oligonucleotide comprising aninosine and/or an uracile and/or a nucleotide containing a base able to form a wobble base pair, said oligonucleotide being preferably RNAse H substantially independent and being complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to a modified oligonucleotide which can be applied in a method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.
摘要翻译： 本发明提供了应用包含肌氨酸和/或尿嘧啶的寡核苷酸和/或含有能够形成摆动碱基对的碱基的核苷酸的方法和药物，所述寡核苷酸优选为RNA酶H，其基本上独立且仅与重复性 在人基因转录物中的序列，用于制备用于诊断，治疗或预防人类中与顺式元件重复不稳定相关的遗传疾病的药物。 因此，本发明提供了一种治疗与顺式元件重复不稳定相关的遗传疾病的方法。 本发明还涉及可以在本发明的方法中应用的修饰的寡核苷酸，以防止细胞中重复扩增的转录物的积累和/或翻译。
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