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公开(公告)号：US08642753B2
公开(公告)日：2014-02-04
申请号：US13566642
申请日：2012-08-03
申请人： Susan M. Freier , Kenneth W. Dobie , Robert McKay , Sanjay Bhanot
发明人： Susan M. Freier , Kenneth W. Dobie , Robert McKay , Sanjay Bhanot
IPC分类号： C07H21/04
CPC分类号： C12N15/1138 , C07K14/705 , C07K14/723 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525
摘要： Compounds, compositions and methods are provided for modulating the expression of glucagon receptor. The compositions comprise oligonucleotides, targeted to nucleic acid encoding glucagon receptor. Methods of using these compounds for modulation of glucagon receptor expression and for diagnosis and treatment of disease associated with expression of glucagon receptor are provided.
摘要翻译： 提供化合物，组合物和方法用于调节胰高血糖素受体的表达。 组合物包含针对编码胰高血糖素受体的核酸的寡核苷酸。 提供了使用这些化合物调节胰高血糖素受体表达和用于诊断和治疗与胰高血糖素受体表达相关的疾病的方法。
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2.发明申请公开(公告)号：US20130109849A1
公开(公告)日：2013-05-02
申请号：US13619362
申请日：2012-09-14
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , A61K31/202 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3521
摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of ACC1 or ACC2 or both in a cell, tissue or animal. 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： 本文公开了用于在细胞，组织或动物中调节ACC1或ACC2或两者的表达的化合物，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的药物中的用途。
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公开(公告)号：US08202981B2
公开(公告)日：2012-06-19
申请号：US13009626
申请日：2011-01-19
申请人： Robert McKay , Ravi Jain , Kenneth W. Dobie , Sanjay K. Pandey , Sanjay Bhanot
发明人： Robert McKay , Ravi Jain , Kenneth W. Dobie , Sanjay K. Pandey , Sanjay Bhanot
CPC分类号： C12N15/1137 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12Y301/03048 , C12N2310/3525
摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of PTPRU in a cell, tissue or animal. Also provided are methods of active target segment validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Also provided are methods for the prevention, amelioration and/or treatment of diabetes, obesity, insulin resistance, insulin deficiency, hypercholesterolemia, hyperglycemia, hyperlipidemia, hypertriglyceridemia, hyperfattyacidemia, liver steatosis, steatohepatitis, non-alcoholic steatohepatitis, metabolic syndrome, cardiovascular disease and coronary heart disease by administration of antisense compounds targeted to PTPRU.
摘要翻译： 本文公开了用于调节细胞，组织或动物中PTPRU表达的化合物，组合物和方法。 还提供了主动目标段验证的方法。 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。 还提供了用于预防，改善和/或治疗糖尿病，肥胖症，胰岛素抵抗，胰岛素缺乏，高胆固醇血症，高血糖症，高脂血症，高甘油三酯血症，高脂肪肝，肝脂肪变性，脂肪性肝炎，非酒精性脂肪性肝炎，代谢综合征，心血管疾病和 通过给予针对PTPRU的反义化合物的冠心病。
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公开(公告)号：US08188059B2
公开(公告)日：2012-05-29
申请号：US12299609
申请日：2007-05-07
申请人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
发明人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
摘要翻译： 本公开描述了短反义化合物，包括长度为化学修饰的高亲和力单体8-16单体的这类化合物。 某些这样的短反义化合物可用于在增加的效力和改善的治疗指数的细胞，组织和动物中还原靶核酸和/或蛋白质。 因此，本文提供了包含用于在体内降低靶RNA的高亲和力核苷酸修饰的短反义化合物。 这样的短反义化合物在比以前描述的反义化合物低的剂量下是有效的，允许降低毒性和治疗成本。 此外，所述短反义化合物具有更大的口服给药潜力。
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公开(公告)号：US08076306B2
公开(公告)日：2011-12-13
申请号：US11734562
申请日：2007-04-12
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525
摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of hepcidin in a cell, tissue or animal or preventing, ameliorating or treating anemia. Also provided are methods for prevention, amelioration or treatment of anemia, and for increasing red blood cell count in an animal. Also provided are methods for the prevention, amelioration and/or treatment of low serum iron levels, low red blood cell count and other clinical endpoints of anemia in an animal. These methods may be achieved by administration of compounds or compositions including antisense compounds targeted to a nucleic acid that expresses hepcidin polypeptide combined with an erythropoiesis stimulating agent.
摘要翻译： 本文公开了用于调节细胞，组织或动物中铁调素表达或预防，改善或治疗贫血的化合物，组合物和方法。 还提供了用于预防，改善或治疗贫血以及用于增加动物中红细胞计数的方法。 还提供了用于预防，改善和/或治疗动物体内低血清铁水平，低红细胞计数和贫血的其它临床终点的方法。 这些方法可以通过施用化合物或组合物来实现，所述化合物或组合物包括靶向表达与促红细胞生成刺激剂组合的铁调素多肽的核酸的反义化合物。
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公开(公告)号：US20110112171A1
公开(公告)日：2011-05-12
申请号：US13009626
申请日：2011-01-19
申请人： Robert McKay , Ravi Jain , Kenneth W. Dobie , Sanjay K. Pandey , Sanjay Bhanot
发明人： Robert McKay , Ravi Jain , Kenneth W. Dobie , Sanjay K. Pandey , Sanjay Bhanot
IPC分类号： A61K31/7088 , A61P3/10 , A61P3/06 , A61P3/04 , A61P9/00
CPC分类号： C12N15/1137 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12Y301/03048 , C12N2310/3525
摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of PTPRU in a cell, tissue or animal. Also provided are methods of active target segment validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Also provided are methods for the prevention, amelioration and/or treatment of diabetes, obesity, insulin resistance, insulin deficiency, hypercholesterolemia, hyperglycemia, hyperlipidemia, hypertriglyceridemia, hyperfattyacidemia, liver steatosis, steatohepatitis, non-alcoholic steatohepatitis, metabolic syndrome, cardiovascular disease and coronary heart diseaseby administration of antisense compounds targeted to PTPRU.
摘要翻译： 本文公开了用于调节细胞，组织或动物中PTPRU表达的化合物，组合物和方法。 还提供了主动目标段验证的方法。 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。 还提供了用于预防，改善和/或治疗糖尿病，肥胖症，胰岛素抵抗，胰岛素缺乏，高胆固醇血症，高血糖症，高脂血症，高甘油三酯血症，高脂肪肝，肝脂肪变性，脂肪性肝炎，非酒精性脂肪性肝炎，代谢综合征，心血管疾病和 通过给予靶向PTPRU的反义化合物的冠心病。
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公开(公告)号：US07919476B2
公开(公告)日：2011-04-05
申请号：US11697280
申请日：2007-04-05
申请人： Sanjay Bhanot , Lynnetta Watts , Robert McKay , Susan M. Freier
发明人： Sanjay Bhanot , Lynnetta Watts , Robert McKay , Susan M. Freier
IPC分类号： A61K48/00
CPC分类号： C12N15/1138 , C07K14/705 , C07K14/723 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525
摘要： Compounds, compositions and methods are provided for modulating the expression of glucagon receptor. The compositions comprise oligonucleotides, targeted to nucleic acid encoding glucagon receptor. Methods of using these compounds for modulation of glucagon receptor expression and for diagnosis and treatment of disease associated with expression of glucagon receptor are provided.
摘要翻译： 提供化合物，组合物和方法用于调节胰高血糖素受体的表达。 组合物包含针对编码胰高血糖素受体的核酸的寡核苷酸。 提供了使用这些化合物调节胰高血糖素受体表达和用于诊断和治疗与胰高血糖素受体表达相关的疾病的方法。
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公开(公告)号：US20110065775A1
公开(公告)日：2011-03-17
申请号：US12883049
申请日：2010-09-15
申请人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
发明人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
IPC分类号： A61K31/7052 , C07H21/00 , A61P3/00 , A61P3/04 , A61P3/10
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
摘要翻译： 本公开描述了短反义化合物，包括长度为化学修饰的高亲和力单体8-16单体的这类化合物。 某些这样的短反义化合物可用于在增加的效力和改善的治疗指数的细胞，组织和动物中还原靶核酸和/或蛋白质。 因此，本文提供了包含用于在体内降低靶RNA的高亲和力核苷酸修饰的短反义化合物。 这样的短反义化合物在比以前描述的反义化合物低的剂量下是有效的，允许降低毒性和治疗成本。 此外，所述短反义化合物具有更大的口服给药潜力。
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公开(公告)号：US20090326042A1
公开(公告)日：2009-12-31
申请号：US12299768
申请日：2007-05-07
申请人： Sanjay Bhanot , Richard Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewitz
发明人： Sanjay Bhanot , Richard Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewitz
IPC分类号： A61K31/7052 , C07H21/00 , C12N5/00 , A61P9/00
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
摘要翻译： 本公开描述了短反义化合物，包括长度为化学修饰的高亲和力单体8-16单体的这类化合物。 某些这样的短反义化合物可用于在增加的效力和改善的治疗指数的细胞，组织和动物中还原靶核酸和/或蛋白质。 因此，本文提供了包含用于在体内降低靶RNA的高亲和力核苷酸修饰的短反义化合物。 这样的短反义化合物在比以前描述的反义化合物低的剂量下是有效的，允许降低毒性和治疗成本。 此外，所述短反义化合物具有更大的口服给药潜力。
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公开(公告)号：US20090306005A1
公开(公告)日：2009-12-10
申请号：US12299572
申请日：2007-05-07
申请人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric Swayze , Edward Wancewicz
发明人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric Swayze , Edward Wancewicz
IPC分类号： A61K31/7088 , C07H21/00 , C12N5/02
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
摘要翻译： 本公开描述了短反义化合物，包括长度为化学修饰的高亲和力单体8-16单体的这类化合物。 某些这样的短反义化合物可用于在增加的效力和改善的治疗指数的细胞，组织和动物中还原靶核酸和/或蛋白质。 因此，本文提供了包含用于在体内降低靶RNA的高亲和力核苷酸修饰的短反义化合物。 这样的短反义化合物在比以前描述的反义化合物低的剂量下是有效的，允许降低毒性和治疗成本。 此外，所述短反义化合物具有更大的口服给药潜力。
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