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1. 发明申请

US20120252879A1 MODULATION OF HUNTINGTIN EXPRESSION 有权
标题翻译： HUNTINGTIN表达的调制
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。

2. 发明授权

US08906873B2 Modulation of huntingtin expression 有权
标题翻译：调节亨廷顿蛋白的表达
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。

3. 发明授权

US08980853B2 Compositions and methods for modulation of SMN2 splicing in a subject 有权
标题翻译：用于调制受试者SMN2拼接的组合物和方法
公开(公告)号：US08980853B2
公开(公告)日：2015-03-17
申请号：US13380021
申请日：2010-06-17
申请人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
发明人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
IPC分类号： A61K31/70 , C07H21/02 , C07H21/04 , A61K31/7088 , C12N15/113 , C12Q1/68 , A61K31/712
CPC分类号： A61K31/7088 , A61K31/712 , C12N15/113 , C12N2310/11 , C12N2320/33
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
摘要翻译：本文公开了用于调节受试者中SMN2 mRNA的剪接的化合物，组合物和方法。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（包括脊髓性肌肉萎缩）的药物中的用途。

4. 发明申请

US20120190728A1 COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT 有权
标题翻译： SMN2在主体中的分离调控的组成和方法
公开(公告)号：US20120190728A1
公开(公告)日：2012-07-26
申请号：US13380021
申请日：2010-06-17
申请人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
发明人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
IPC分类号： A61K31/713 , A61P25/28
CPC分类号： A61K31/7088 , A61K31/712 , C12N15/113 , C12N2310/11 , C12N2320/33
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
摘要翻译：本文公开了用于调节受试者中SMN2 mRNA的剪接的化合物，组合物和方法。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（包括脊髓性肌肉萎缩）的药物中的用途。

5. 发明申请

US20100081705A1 METHODS FOR SLOWING FAMILIAL ALS DISEASE PROGRESSION 审中-公开
标题翻译：缓解家系ALS疾病进展的方法
公开(公告)号：US20100081705A1
公开(公告)日：2010-04-01
申请号：US12566549
申请日：2009-09-24
申请人： C. Frank Bennett , Don W. Cleveland , Richard Alan Smith , Susan M. Freier , Thomas P. Condon
发明人： C. Frank Bennett , Don W. Cleveland , Richard Alan Smith , Susan M. Freier , Thomas P. Condon
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。

6. 发明申请

US20130237585A1 MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION 审中-公开
标题翻译： DYSTROPHIA MYOTONICA - 蛋白激酶（DMPK）表达的调控
公开(公告)号：US20130237585A1
公开(公告)日：2013-09-12
申请号：US13811181
申请日：2011-07-19
申请人： C. Frank Bennett , Susan M. Freier , Robert A. MacLeod , Sanjay K. Pandey , Charles A. Thornton , Thurman Wheeler , Seng H. Cheng , Andrew Leger , Bruce M. Wentworth
发明人： C. Frank Bennett , Susan M. Freier , Robert A. MacLeod , Sanjay K. Pandey , Charles A. Thornton , Thurman Wheeler , Seng H. Cheng , Andrew Leger , Bruce M. Wentworth
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/3181 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525 , C12Y207/11 , C12N2310/322 , C12N2310/3533 , C12N2310/3527
摘要： Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
摘要翻译：本文提供了用于减少动物体内DMPK mRNA和蛋白质表达的方法，化合物和组合物。本文还提供了用于优先减少CUGexp DMPK RNA，减少肌动蛋白或减少动物中的拼接病变的方法，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1型肌强直营养不良症或其症状。

7. 发明授权

US08669102B2 Modulation of prion expression 有权
标题翻译：调节朊病毒表达
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
IPC分类号： C12N15/85 , C07H21/04 , C07H21/02
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）。

8. 发明申请

US20100069472A1 COMPOSITIONS AND THEIR USES DIRECTED TO HUNTINGTIN 审中-公开
标题翻译：组合物及其与HUNTINGTIN有关的用途
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。

9. 发明授权

US08921331B2 Methods for slowing familial ALS disease progression 有权
标题翻译：减慢家族性ALS疾病进展的方法
公开(公告)号：US08921331B2
公开(公告)日：2014-12-30
申请号：US13406256
申请日：2012-02-27
申请人： C. Frank Bennett , Susan M. Freier , Kenneth W. Dobie
发明人： C. Frank Bennett , Susan M. Freier , Kenneth W. Dobie
IPC分类号： A61K48/00 , C12N15/113 , A61K38/00
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。

10. 发明授权

US08791088B2 Compositions and methods for modulation of LMNA expression 有权
标题翻译： LMNA表达调控的组成和方法
公开(公告)号：US08791088B2
公开(公告)日：2014-07-29
申请号：US13566922
申请日：2012-08-03
申请人： C. Frank Bennett , Kenneth W. Dobie , Susan M. Freier , Stanley T. Crooke
发明人： C. Frank Bennett , Kenneth W. Dobie , Susan M. Freier , Stanley T. Crooke
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2320/33
摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.
摘要翻译：本文公开了用于调节细胞，组织或动物中LMNA表达的化合物，组合物和方法。还提供了目标验证的方法。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。还提供了使用所公开的化合物鉴定所选mRNA的顺式剪接调节元件的方法。

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