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11.发明申请RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) 有权使用化学修饰的短干扰核酸（siNA）的RNA干扰介导的基因表达抑制公开(公告)号：US20070004664A1
公开(公告)日：2007-01-04
申请号：US11499529
申请日：2006-08-04
申请人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
发明人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
IPC分类号： A61K48/00 , C07H21/02 , C07F9/6512
CPC分类号： C12N15/111 , A61K9/0019 , A61K9/0048 , A61K47/544 , A61K47/549 , A61K47/554 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/351 , C12N2320/51 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
摘要翻译： 本发明涉及可用于调节各种应用中的基因表达的方法和试剂，包括在治疗，诊断，靶标验证和基因组发现应用中的用途。 具体地，本发明涉及合成的化学修饰的小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA （shRNA）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。 小核酸分子可用于治疗对细胞，组织或生物中基因表达或活性的调节作出反应的任何疾病或病症。
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12.发明申请公开(公告)号：US20060293272A1
公开(公告)日：2006-12-28
申请号：US11499533
申请日：2006-08-04
申请人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
发明人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
CPC分类号： C07F9/65616 , A61K9/0019 , A61K47/544 , A61K47/549 , A61K47/554 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/351 , C12N2320/51 , C12N2330/30 , Y02A50/465 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
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13.发明申请公开(公告)号：US20060292691A1
公开(公告)日：2006-12-28
申请号：US11502885
申请日：2006-08-11
申请人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
发明人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
IPC分类号： A61K48/00 , C07H21/02 , C07F9/6512
CPC分类号： C07F9/65616 , A61K9/0019 , A61K47/544 , A61K47/549 , A61K47/554 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/351 , C12N2320/51 , C12N2330/30 , Y02A50/465 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
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14.发明申请公开(公告)号：US20060275903A1
公开(公告)日：2006-12-07
申请号：US11502893
申请日：2006-08-11
申请人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
发明人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
CPC分类号： C07F9/65616 , A61K9/0019 , A61K47/544 , A61K47/549 , A61K47/554 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/351 , C12N2320/51 , C12N2330/30 , Y02A50/465 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
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公开(公告)号：US20060036090A1
公开(公告)日：2006-02-16
申请号：US11251407
申请日：2005-10-14
CPC分类号： C03C17/28 , C03C17/3405 , C07B2200/11 , C07F7/1804 , C07H21/04 , G01N33/54353 , G01N33/552 , Y10S530/811
摘要： The present invention features novel compositions, linkers, derivatized solid supports, and methods for the efficient solid phase synthesis of oligonucleotides, including RNA, DNA, RNA-DNA chimeras, and analogs thereof.
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公开(公告)号：US06995259B1
公开(公告)日：2006-02-07
申请号：US09887182
申请日：2001-06-22
IPC分类号： C07H21/00 , C07H19/04 , C12N11/14 , G01N33/551 , C07K1/00
CPC分类号： C03C17/28 , C03C17/3405 , C07B2200/11 , C07F7/1804 , C07H21/04 , G01N33/54353 , G01N33/552 , Y10S530/811
摘要： The present invention features novel compositions, linkers, derivatized solid supports, and methods for the efficient solid phase synthesis of oligonucleotides, including RNA, DNA, RNA-DNA chimeras, and analogs thereof.
摘要翻译： 本发明的特征在于新颖的组合物，接头，衍生的固体支持物以及有效固相合成寡核苷酸的方法，包括RNA，DNA，RNA-DNA嵌合体及其类似物。
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公开(公告)号：US5077403A
公开(公告)日：1991-12-31
申请号：US568412
申请日：1990-08-16
申请人： Elie Abushanab , Chandra Vargeese
发明人： Elie Abushanab , Chandra Vargeese
摘要： A process for the production of a compound of the formula ##STR1## where R.sub.1 and R.sub.2 each are alkyl C.sub.1 -C.sub.16, aryl, aralkyl, alkenyl, alkynyl, cyano, carboxy and esters, carboximido, N-monosubstituted and N,N-disubstituted carboximido with alkyl, aralkyl, and aryl groups; R.sub.3 is tertiary butyldimethyl silyl, R.sub.4 is tertiarybutyldimethylsilyl, trimethylsilyl, X=O or NH comprising condensing a compound of the formula ##STR2## wherein R.sub.3 and R.sub.4 are as defined above with a compound of either the formula: ##STR3## wherein R.sub.1 and R.sub.2 are as defined above and R.sub.5 is C.sub.1 -C.sub.4 ; in the presence of a reaction-inert solvent at a temperature of from 50.degree.to about 150.degree. C. forming an intermediate and oxidizing the intermediate product, to form the desired compound of formula I. The products are antiviral.
摘要翻译： 制备式Ⅰ化合物的方法，其中R 1和R 2各自为C 1 -C 16烷基，芳基，芳烷基，烯基，炔基，氰基，羧基和酯，碳代亚氨基，N-单取代和N，N-二 具有烷基，芳烷基和芳基的二取代的羰基亚氨基; R3是叔丁基二甲基甲硅烷基，R4是叔丁基二甲基甲硅烷基，三甲基甲硅烷基，X = O或NH，其包括将下式化合物（其中R 3和R 4如上定义）与下式化合物缩合： 图像IIIb其中R1和R2如上定义，R5是C1-C4; 在反应惰性溶剂存在下，在50至150℃的温度下形成中间体并氧化中间产物，形成所需的式I化合物。产物是抗病毒的。
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公开(公告)号：US20220306573A1
公开(公告)日：2022-09-29
申请号：US17046752
申请日：2019-04-11
申请人： Jason Jingxi ZHANG , Chandra VARGEESE , Naoki IWAMOTO , Chikdu Shakti SHIVALILA , Nayantara KOTHARI , Ann Fiegen DURBIN , Selvi RAMASAMY , Pachamuthu KANDASAMY , Jayakanthan KUMARASAMY , Gopal Reddy BOMMINENI , Subramanian MARAPPAN , Sethumadhavan DIVAKARAMENON , David Charles Donnel BUTLER , Genliang LU , Hailin YANG , Mamoru SHIMIZU , Prashant MONIAN , WAVE LIFE SCIENCES LTD.
发明人： Jason Jingxin Zhang , Chandra Vargeese , Naoki Iwamoto , Chikdu Shakti Shivalila , Nayantara Kothari , Ann Fiegen Durbin , Selvi Ramasamy , Pachamuthu Kandasamy , Jayakanthan Kumarasamy , Gopal Reddy Bommineni , Subramanian Marappan , Sethumadhavan Divakaramenon , David Charles Donnell Butler , Genliang Lu , Hailin Yang , Mamoru Shimizu , Prashant Monian
IPC分类号： C07C317/28 , C07H21/02 , C07H21/04 , C12N15/113
摘要： Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for reducing levels of transcripts. In some embodiments, the present disclosure provides technologies useful for modulating transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.
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19.发明授权RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) 有权使用化学修饰的短干扰核酸（siNA）的RNA干扰介导的基因表达抑制公开(公告)号：US08846894B2
公开(公告)日：2014-09-30
申请号：US11676124
申请日：2007-02-16
申请人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon F Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra K Vaish
发明人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon F Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra K Vaish
CPC分类号： C07F9/65616 , A61K9/0019 , A61K47/544 , A61K47/549 , A61K47/554 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/351 , C12N2320/51 , C12N2330/30 , Y02A50/465 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
摘要翻译： 本发明涉及可用于调节各种应用中的基因表达的方法和试剂，包括在治疗，诊断，靶标验证和基因组发现应用中的用途。 具体地，本发明涉及合成的化学修饰的小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA （shRNA）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。 小核酸分子可用于治疗对细胞，组织或生物中的基因表达或活性的调节作出反应的任何疾病或病症。
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20.发明授权RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA) 有权使用短干扰核酸（siNA）的RNA干扰介导的丙型肝炎病毒（HCV）表达的抑制公开(公告)号：US07935812B2
公开(公告)日：2011-05-03
申请号：US12418477
申请日：2009-04-03
CPC分类号： C12N15/1131 , A61K47/6911 , C12N2310/14 , C12N2770/24211
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of hepatitis C virus (HCV) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in hepatitis C virus (HCV) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules capable of mediating or that mediate RNA interference (RNAi) against hepatitis C virus (HCV) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules.
摘要翻译： 本发明涉及用于研究，诊断和治疗对丙型肝炎病毒（HCV）基因表达和/或活性的调节作用的性状，疾病和病症的化合物，组合物和方法。 本发明还涉及与涉及丙型肝炎病毒（HCV）基因表达途径或介导的其他细胞过程的基因的表达和/或活性的调节作用的性状，疾病和病症有关的化合物，组合物和方法 维持或发展这些性状，疾病和状况。 具体地，本发明涉及能够介导或介导针对丙型肝炎病毒（HCV）基因表达的RNA干扰（RNAi）的双链核酸分子，包括这种小核酸分子的混合物和这种小的核酸分子的脂质纳米颗粒（LNP）制剂 核酸分子。
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