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公开(公告)号：US20100069472A1
公开(公告)日：2010-03-18
申请号：US12618470
申请日：2009-11-13
申请人： Gene Hung , Janet M. Leeds , C. Frank Bennett , Susan M. Freier
发明人： Gene Hung , Janet M. Leeds , C. Frank Bennett , Susan M. Freier
IPC分类号： A61K31/7088 , C07H21/04 , A61P25/28
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/111 , C12N2310/31 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/33 , C12N2310/3341 , C12N2310/335 , C12N2310/341 , C12N2310/346 , C12N2320/30 , C12N2310/3525
摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.
摘要翻译： 本文公开了用于调节细胞，组织或动物中亨廷丁氏蛋白的表达的化合物，组合物和方法。 还提供了使用靶向亨廷顿汀的反义化合物减慢或预防亨廷顿病进展的方法。 另外提供的方法是延缓或预防在亨廷顿病易感的个体中亨廷顿病发病的方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症的药物中的用途。
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公开(公告)号：US20120252879A1
公开(公告)日：2012-10-04
申请号：US13395188
申请日：2010-09-10
申请人： Gene Hung , C. Frank Bennett , Susan M. Freier , Holly Kordasiewicz , Lisa Stanek , Don W. Cleveland , Seng H. Cheng , Lamya Shihabuddin
发明人： Gene Hung , C. Frank Bennett , Susan M. Freier , Holly Kordasiewicz , Lisa Stanek , Don W. Cleveland , Seng H. Cheng , Lamya Shihabuddin
IPC分类号： A61K31/711 , A61P25/14
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/345 , C12N2310/346 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
摘要翻译： 本文提供了用于降低动物中亨廷丁蛋白mRNA和蛋白质表达的方法，化合物和组合物。 这些方法，化合物和组合物可用于治疗，预防，延迟或改善亨廷顿病或其症状。
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公开(公告)号：US08669102B2
公开(公告)日：2014-03-11
申请号：US13059058
申请日：2009-08-14
申请人： C. Frank Bennett , Gene Hung , Susan M. Freier , Kenneth W. Dobie
发明人： C. Frank Bennett , Gene Hung , Susan M. Freier , Kenneth W. Dobie
CPC分类号： A61K31/7125
摘要： Disclosed herein are compounds and methods for decreasing PrP and preventing, ameliorating, or treating a prion disease or conformational neurodegenerative disorder, in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to PrP include Creutzfeldt-Jakob disease (CJD); variant Creutzfeldt-Jakob Disease (vCJD); Gerstmann-Straussler-Scheinker syndrome; fatal familial insomnia; kuru; Bovine Spongiform Encephalopathy (BSE), e.g. “mad cow disease”; Chronic Wasting Disease (CWD); scrapie; transmissible mink encephalopathy; feline spongiform encephalopathy; ungulate spongiform encephalopathy; Alzheimer's disease; Parkinson's disease; Huntington's disease; and Amyotrophic Lateral Sclerosis (ALS).
摘要翻译： 本文公开了在有需要的个体中减少PrP并预防，改善或治疗朊病毒疾病或构象神经变性疾病的化合物和方法。 通过施用针对PrP的反义化合物可以改善的疾病状况的实例包括克雅氏病（CJD）; 变异克雅氏病（vCJD）; Gerstmann-Straussler-Scheinker综合征; 致命性家族性失眠; 库鲁 牛海绵状脑病（BSE），例如。 “疯牛病”; 慢性疾病（CWD）; scrap子 传染性貂脑病 猫海绵状脑病; 有蹄型海绵状脑病 阿尔茨海默氏病; 帕金森病 亨廷顿氏病 和肌萎缩性侧索硬化（ALS）。
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公开(公告)号：US08906873B2
公开(公告)日：2014-12-09
申请号：US13395188
申请日：2010-09-10
申请人： Gene Hung , C. Frank Bennett , Susan M. Freier , Holly Kordasiewicz , Lisa Stanek , Don W. Cleveland , Seng H. Cheng , Lamya Shihabuddin
发明人： Gene Hung , C. Frank Bennett , Susan M. Freier , Holly Kordasiewicz , Lisa Stanek , Don W. Cleveland , Seng H. Cheng , Lamya Shihabuddin
IPC分类号： A61K48/00 , C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/345 , C12N2310/346 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
摘要翻译： 本文提供了用于降低动物中亨廷丁蛋白mRNA和蛋白质表达的方法，化合物和组合物。 这些方法，化合物和组合物可用于治疗，预防，延迟或改善亨廷顿病或其症状。
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5.发明授权Oligomeric compounds and compositions for use in modulation of small non-coding RNAs 有权用于调节小非编码RNA的低聚化合物和组合物公开(公告)号：US08946179B2
公开(公告)日：2015-02-03
申请号：US13412307
申请日：2012-03-05
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/14 , C12N2310/141 , C12N2310/31 , C12N2310/315 , C12N2310/317 , C12N2310/3181 , C12N2310/32 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/11 , C12N2320/30 , C12N2320/50 , C12N2310/3525
摘要： Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.
摘要翻译： 提供化合物，组合物和方法用于调节小非编码RNA的表达和功能。 组合物包含靶向小的非编码RNA的寡聚化合物。 还提供了使用这些化合物调节小非编码RNA以及这些RNA的下游靶标以及用于诊断和治疗与小非编码RNA相关的疾病的方法。
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公开(公告)号：US20110172296A1
公开(公告)日：2011-07-14
申请号：US13005508
申请日：2011-01-12
IPC分类号： A61K31/7088 , C07H21/04 , C07H21/02 , A61P17/02
CPC分类号： A61K31/7088 , C12N15/1136 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3521 , C12N2310/3525
摘要： Provided are compounds capable of inhibiting expression of TGF-beta 1 and compositions containing same as well as methods using such compounds for treating fibrotic diseases including the reduction of scarring resulting from wound healing.
摘要翻译： 提供能够抑制TGF-β1的表达的化合物及其含有组合物的化合物以及使用这些化合物治疗纤维化疾病的方法，包括减少由伤口愈合导致的瘢痕形成。
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公开(公告)号：US20110160283A1
公开(公告)日：2011-06-30
申请号：US12743797
申请日：2008-11-20
IPC分类号： A61K31/7088 , C07H21/04 , A61P35/00 , A61P29/00
CPC分类号： C12N15/113 , C12N15/1138 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525
摘要： Disclosed herein are antisense compounds and methods for decreasing CD40. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to CD40 include hyperproliferative disorders, graft versus host disease (GVHD), graft rejection, asthma, airway hyperresponsiveness, chronic obstructive pulmonary disease (COPD), multiple sclerosis (MS), systemic lupus erythematosus (SLE), and certain forms of arthritis.
摘要翻译： 本文公开了用于减少CD40的反义化合物和方法。 通过施用靶向CD40的反义化合物可以改善的疾病状况的实例包括过度增殖性疾病，移植物抗宿主病（GVHD），移植物排斥反应，哮喘，气道高反应性，慢性阻塞性肺疾病（COPD），多发性硬化（MS） ，系统性红斑狼疮（SLE）和某些形式的关节炎。
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公开(公告)号：US07902163B2
公开(公告)日：2011-03-08
申请号：US11449446
申请日：2006-06-07
CPC分类号： C12N15/1137 , A61K38/00 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525 , C12Y115/01001 , Y02P20/582
摘要： Antisense compounds, compositions and methods are provided for modulating the expression of superoxide dismutase 1, soluble. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding superoxide dismutase 1, soluble. Methods of using these compounds for modulation of superoxide dismutase 1, soluble expression and for treatment of diseases associated with expression of superoxide dismutase 1, soluble are provided.
摘要翻译： 提供反义化合物，组合物和方法用于调节超氧化物歧化酶1的表达，可溶性。 组合物包含靶向编码超氧化物歧化酶1的核酸的可反应化合物，特别是反义寡核苷酸。 提供了使用这些化合物调节超氧化物歧化酶1，可溶性表达和治疗与超氧化物歧化酶1表达相关的疾病的方法。
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9.发明申请Oligomeric Compounds And Compositions For Use In Modulation Of Small Non-Coding RNAs 有权用于调节小非编码RNA的低聚化合物和组合物公开(公告)号：US20100267813A1
公开(公告)日：2010-10-21
申请号：US12346940
申请日：2008-12-31
申请人： Christine Esau , Bridget Lollo , C. Frank Bennett , Susan M. Freier , Richard H. Griffey , Brenda F. Baker , Timothy A. Vickers , Eric G. Marcusson , Erich Koller , Eric E. Swayze , Ravi Jain , Balkrishen Bhat , Eigen Peralta
发明人： Christine Esau , Bridget Lollo , C. Frank Bennett , Susan M. Freier , Richard H. Griffey , Brenda F. Baker , Timothy A. Vickers , Eric G. Marcusson , Erich Koller , Eric E. Swayze , Ravi Jain , Balkrishen Bhat , Eigen Peralta
IPC分类号： A61K31/7088 , C07H21/02
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/14 , C12N2310/141 , C12N2310/31 , C12N2310/315 , C12N2310/317 , C12N2310/3181 , C12N2310/32 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/11 , C12N2320/30 , C12N2320/50 , C12N2310/3525
摘要： Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.
摘要翻译： 提供化合物，组合物和方法用于调节小非编码RNA的表达和功能。 组合物包含靶向小的非编码RNA的寡聚化合物。 还提供了使用这些化合物调节小非编码RNA以及这些RNA的下游靶标以及用于诊断和治疗与小非编码RNA相关的疾病的方法。
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公开(公告)号：US20100081705A1
公开(公告)日：2010-04-01
申请号：US12566549
申请日：2009-09-24
IPC分类号： A61K31/7088 , A61P25/00
CPC分类号： C12N15/1137 , A61K38/00 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12Y115/01001 , Y02P20/582 , C12N2310/3525
摘要： Methods for slowing disease progression in an individual suffering from familial ALS are provided. Also provided are methods of increasing the survival time of an individual suffering from familial ALS. These methods employ antisense oligonucleotides targeted to SOD1, for use in inhibiting the expression of SOD1 in the central nervous system of an individual suffering from familial ALS.
摘要翻译： 提供了减轻患有家族性ALS的个体疾病进展的方法。 还提供了增加患有家族性ALS的个体的存活时间的方法。 这些方法使用靶向SOD1的反义寡核苷酸，用于抑制患有家族性ALS的个体的中枢神经系统中SOD1的表达。
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