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公开(公告)号:US12023366B2
公开(公告)日:2024-07-02
申请号:US17000867
申请日:2020-08-24
申请人: GENETHON , ROYAL HOLLOWAY AND BEDFORD NEW COLLEGE , ASSOCIATION INSTITUT DE MYOLOGIE , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , SORBONNE UNIVERSITE , CENTRE HOSPITALIER UNIVERSITAIRE DE NANTES , NANTES UNIVERSITE
CPC分类号: A61K38/1719 , A61K48/005 , A61K48/0058 , A61K48/0075 , C12N7/00 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12N2750/14171 , C12N2840/007
摘要: A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein:
the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin;
the composition is systemically administered.-
公开(公告)号:US10786546B2
公开(公告)日:2020-09-29
申请号:US16185248
申请日:2018-11-09
摘要: A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; the composition is systemically administered.
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公开(公告)号:US20140059089A1
公开(公告)日:2014-02-27
申请号:US13994735
申请日:2011-12-16
IPC分类号: G06F17/30
摘要: There is provided a method of structuring a network of nodes, comprising: providing link information relating to existing links between the nodes (2); using the link information to partition the network into non-predetermined groups of related nodes (3), thereby forming a group structure for the network; identifying for each group a corpus of information associated with the nodes in that group (4); generating for each group a machine-readable characterisation of that group based on the corpus of information identified for the group (5); and structuring the network of nodes through the groups and their associated characterisations (2 to 7).
摘要翻译: 提供了一种构造节点网络的方法,包括:提供与节点(2)之间的现有链路相关的链接信息; 使用链接信息将网络划分成非预定的相关节点组(3),从而形成网络的组结构; 识别每个组中与该组中的节点相关联的信息语料库(4); 基于为组识别的信息语料库(5),为每个组生成该组的机器可读特征; 并通过组及其相关特征构建节点网络(2到7)。
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公开(公告)号:US20120065244A1
公开(公告)日:2012-03-15
申请号:US13204326
申请日:2011-08-05
IPC分类号: A61K31/7088 , C12N15/63 , C07H21/04
CPC分类号: C12N15/113 , A61K31/7088 , C12N15/115 , C12N2310/11 , C12N2310/321 , C12N2310/3233 , C12N2310/351 , C12N2310/3513 , C12N2320/31 , C12N2320/35
摘要: Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.
摘要翻译: 提供分子用于诱导或促进外显子跳跃以在细胞中从mRNA前分子形成剪接的mRNA产物。 分子可以直接作为寡核苷酸提供,或者被施用于受试者的载体的表达产物。 可以实现高跳脱率。 跳高率降低了像Duchene肌营养不良这样的疾病的严重程度,使得该疾病更像是Becker肌营养不良症。 这是症状严重程度和死亡率的严重降低。
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公开(公告)号:US20230098909A1
公开(公告)日:2023-03-30
申请号:US18050217
申请日:2022-10-27
IPC分类号: G01K7/30
摘要: A current sensing noise thermometer comprising: a sensor resistor thermally coupled to a target to be measured; a superconducting coil; superconducting thermal breaks between respective ends of the sensor resistor and respective ends of the superconducting coil; and a superconducting flux sensor; wherein the sensor resistor, superconducting coil and superconducting thermal breaks form a loop inductively coupled to the superconducting flux sensor. There may be a noise filter between the sensor resistor and the superconducting coil.
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公开(公告)号:US20220195438A1
公开(公告)日:2022-06-23
申请号:US17405439
申请日:2021-08-18
IPC分类号: C12N15/113 , A61K47/54 , C07H21/04
摘要: Certain disclosed oligomers induce exon skipping during processing of myostatin pre-mRNA. The oligomers may be in a vector or encoded by the vector. The vector is used for inducing exon skipping during processing of myostatin pre-mRNA. A therapeutically effective amount of the oligomer may be administered to a subject patient such that exon skipping during processing of myostatin pre-mRNA is induced. The administration to a subject may be used in order to increase or maintain muscle mass, or slowing degeneration of muscle mass in the subject. The administration to a subject may ameliorate muscle wasting conditions, such as muscular dystrophy. Examples of such muscular dystrophies which may be so treated include Becker's muscular dystrophy, congenital muscular dystrophy, Duchenne muscular dystrophy, distal muscular dystrophy, Emery-Dreifuss muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), limb-girdle muscular dystrophy, myotonic muscular dystrophy, and oculopharyngeal muscular dystrophy.
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公开(公告)号:US20210130426A1
公开(公告)日:2021-05-06
申请号:US16770358
申请日:2018-12-05
发明人: George Dickson , Linda Popplewell
摘要: There is described a nucleic acid molecule comprising a nucleotide sequence encoding a functional dystrophin protein. Also described is a vector, a host cell and a pharmaceutical composition comprising the nucleic acid molecule; use of the nucleic acid molecule in therapy, such as in the treatment of a muscular dystrophy; and a method of treating muscular dystrophy, the method comprising administering a therapeutically effective amount of the nucleic acid molecule to a patient suffering from a muscular dystrophy.
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公开(公告)号:US09139625B2
公开(公告)日:2015-09-22
申请号:US14118677
申请日:2012-05-10
IPC分类号: C07K16/00 , C12P21/08 , C07K14/33 , C12Q1/04 , G01N33/569 , A61K39/07 , A61K39/08 , C07K16/12 , A61K39/00
CPC分类号: C07K14/33 , A61K39/07 , A61K39/08 , A61K2039/523 , A61K2039/542 , A61K2039/70 , C07K16/1282 , C12Q1/04 , G01N33/56911 , G01N2333/32 , G01N2333/33 , Y02A50/469
摘要: The invention provides novel surface coat proteins of Clostridium spp. spores, and nucleic acids encoding such proteins. The invention extends to various uses of these nucleic acids and proteins, as antigens for use in vaccine design and construction, and to vaccines per se, and in diagnostic test kits and methods for the detection of Clostridium spp. infections. In addition, the nucleic acids and proteins can be used as potential targets for therapeutic drugs for the prevention or treatment of Clostridium spp. infections.
摘要翻译: 本发明提供了Clostridium spp的新型表面外壳蛋白。 孢子和编码这种蛋白质的核酸。 本发明延伸到这些核酸和蛋白质的各种用途,作为用于疫苗设计和构建的抗原以及疫苗本身以及用于检测梭菌的诊断测试试剂盒和方法。 感染。 此外,核酸和蛋白质可以用作预防或治疗梭菌属的治疗药物的潜在靶标。 感染。
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公开(公告)号:US20130085139A1
公开(公告)日:2013-04-04
申请号:US13644363
申请日:2012-10-04
IPC分类号: A61K31/5377 , C12N15/85 , A61K31/713 , A61P21/00 , C07H21/04 , C07D473/34
摘要: Certain disclosed oligomers induce exon skipping during processing of myostatin pre-mRNA. The oligomers may be in a vector or encoded by the vector. The vector is used for inducing exon skipping during processing of myostatin pre-mRNA. A therapeutically effective amount of the oligomer may be administered to a subject patient such that exon skipping during processing of myostatin pre-mRNA is induced. The administration to a subject may be used in order to increase or maintain muscle mass, or slowing degeneration of muscle mass in the subject. The administration to a subject may ameliorate muscle wasting conditions, such as muscular dystrophy. Examples of such muscular dystrophies which may be so treated include Becker's muscular dystrophy, congenital muscular dystrophy, Duchenne muscular dystrophy, distal muscular dystrophy, Emery-Dreifuss muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), limb-girdle muscular dystrophy, myotonic muscular dystrophy, and oculopharyngeal muscular dystrophy
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10.
公开(公告)号:US20120237544A1
公开(公告)日:2012-09-20
申请号:US13497169
申请日:2010-09-21
IPC分类号: A61K35/74 , A61K39/21 , A61K39/145 , A61K39/245 , A61K39/205 , A61K39/108 , A61K39/07 , A61K39/095 , A61K39/02 , A61K39/05 , A61K39/112 , A61K39/04 , A61K39/002 , A61K39/008 , A61K39/015 , A61P37/04 , A61K39/00
CPC分类号: A61K35/74 , A61K39/0003 , A61K39/015 , A61K39/07 , A61K39/08 , A61K39/385 , A61K2039/542 , A61K2039/543 , Y02A50/41 , Y02A50/412 , Y02A50/478 , Y02A50/489
摘要: The present invention relates to a method of coating a spore with one or more therapeutic agents. The present invention also relates to a coated spore obtained by the method of the present invention and the use of the coated spore as a vaccine.
摘要翻译: 本发明涉及用一种或多种治疗剂包衣孢子的方法。 本发明还涉及通过本发明的方法获得的包被的孢子以及涂层孢子作为疫苗的用途。
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