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1. 发明申请

US20080039412A1 Rna Interference Mediated Inhibition of Gene Expression Using Multifunctional Short Interfering Nucleic Acid (Multifunctional Sina) 有权
标题翻译： Rna干扰介导的多功能短干扰核酸基因表达抑制（多功能新浪）
公开(公告)号：US20080039412A1
公开(公告)日：2008-02-14
申请号：US10597755
申请日：2005-02-09
申请人： Vasant Jadhav , Shawn Zinnen
发明人： Vasant Jadhav , Shawn Zinnen
IPC分类号： A61K31/70 , A61P43/00 , C07H19/00
CPC分类号： C12N15/1131 , A61K38/00 , C12N15/1136 , C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/3519 , C12N2310/51 , C12N2310/53
摘要： The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to multifunctional short interfering nucleic acid (multifunctional siNA) molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism via RNA interference (RNAi). The bifunctional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA 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基于核酸的试剂，包括在治疗，兽医，农业，诊断，靶标验证和基因组发现应用中的用途。具体地，本发明涉及通过RNA干扰（RNAi）调节生物系统例如细胞，组织或生物体中的一种或多种基因的表达的多功能短干扰核酸（多功能siNA）分子。本发明的双功能短干扰核酸（多功能siNA）分子可以靶向单个靶核酸分子中的多于一个核酸序列区域，或者靶向不同靶核酸分子中核酸序列的区域。自身多功能siNA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。

2. 发明申请

US20070032441A1 Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina) 审中-公开
标题翻译： Rna干扰介导使用化学修饰的短干扰核酸（新生）的基因表达抑制
公开(公告)号：US20070032441A1
公开(公告)日：2007-02-08
申请号：US10557542
申请日：2004-05-24
申请人： James McSwiggen , David Morrissey , Shawn Zinnen , Vasant Jadhav , Narendra Vaish
发明人： James McSwiggen , David Morrissey , Shawn Zinnen , Vasant Jadhav , Narendra Vaish
IPC分类号： A61K48/00 , C07H21/02
CPC分类号： C12N15/87 , A61K38/00 , C07H21/02 , C12N15/111 , C12N15/113 , C12N15/1131 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/332 , C12N2310/344 , C12N2310/346 , C12N2310/3515 , C12N2320/32 , C12N2320/51 , C12Y103/01022 , C12Y104/03003 , C12Y114/19001 , C12Y207/07049 , C12Y207/11001 , C12Y207/11013 , C12Y301/03048 , C12Y604/01002 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, cosmetic, cosmeceutical, prophylactic, 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 (e.g., cancer, proliferative, inflammatory, metabolic, autoimmune, neurologic, ocular diseases), condition, trait (e.g., hair growth and removal), genotype or phenotype that responds to modulation of gene expression or activity in a cell, tissue, or organism. Such small nucleic acid molecules can be administered systemically, locally, or topically.
摘要翻译：本发明涉及可用于调节多种应用中的基因表达的方法和试剂，包括用于治疗，化妆品，药妆，预防，诊断，靶标验证和基因组发现应用。具体地说，本发明涉及合成的化学修饰的小核酸分子，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任何疾病（例如癌症，增殖性，炎症，代谢，自身免疫，神经系统，眼部疾病），病症，性状（例如毛发生长和去除），基因型或表型，调节细胞，组织或生物体中的基因表达或活性。这样的小核酸分子可以全身，局部或局部施用。

3. 发明申请

US20110118335A1 RNA Interference Mediated Inhibition Of Gene Expression Using Multifunctional Short Interfering Nucleic Acid (Multifunctional siNA) 审中-公开
标题翻译： RNA干扰介导的使用多功能短干扰核酸（多功能siNA）的基因表达抑制
公开(公告)号：US20110118335A1
公开(公告)日：2011-05-19
申请号：US12947609
申请日：2010-11-16
申请人： Vasant Jadhav , Shawn Zinnen
发明人： Vasant Jadhav , Shawn Zinnen
IPC分类号： A61K31/7105 , C07H21/00 , C07H21/02 , A61K31/7088
CPC分类号： C12N15/1131 , A61K38/00 , C12N15/1136 , C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/3519 , C12N2310/51 , C12N2310/53
摘要： The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to multifunctional short interfering nucleic acid (multifunctional siNA) molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism via RNA interference (RNAi). The bifunctional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA 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基于核酸的试剂，包括在治疗，兽医，农业，诊断，靶标验证和基因组发现应用中的用途。具体地，本发明涉及通过RNA干扰（RNAi）调节生物系统例如细胞，组织或生物体中的一种或多种基因的表达的多功能短干扰核酸（多功能siNA）分子。本发明的双功能短干扰核酸（多功能siNA）分子可以靶向单个靶核酸分子中的多于一个核酸序列区域，或者靶向不同靶核酸分子中核酸序列的区域。自身多功能siNA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。

4. 发明申请

US20050233998A1 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA) 审中-公开
标题翻译： RNA干扰介导的使用短干扰核酸（siNA）抑制血管内皮生长因子和血管内皮生长因子受体基因表达
公开(公告)号：US20050233998A1
公开(公告)日：2005-10-20
申请号：US10944611
申请日：2004-09-16
申请人： Vasant Jadhav , Karl Kossen , Shawn Zinnen , Narendra Vaish , James McSwiggen
发明人： Vasant Jadhav , Karl Kossen , Shawn Zinnen , Narendra Vaish , James McSwiggen
IPC分类号： A01N43/04 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12P19/34
CPC分类号： C12N15/113 , C12N2310/14
摘要： This invention relates to compounds, compositions, and methods useful for modulating VEGF and/or VEGFR gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of VEGF and/or VEGFR gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (mRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of VEGF and/or VEGFR genes.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节VEGF和/或VEGFR基因表达的化合物，组合物和方法。本发明还涉及使用小核酸分子调节参与VEGF和/或VEGFR基因表达和/或活性通过RNA干扰（RNAi）的其它基因的表达和活性的化合物，组合物和方法。特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（mRNA）和短发夹RNA（shRNA））分子和用于调节VEGF和/或VEGFR基因表达的方法。

5. 发明授权

US07858769B2 RNA interference mediated inhibition of gene expression using multifunctional short interfering nucleic acid (multifunctional siNA) 有权
标题翻译：使用多功能短干扰核酸（多功能siNA）的RNA干扰介导的基因表达抑制
公开(公告)号：US07858769B2
公开(公告)日：2010-12-28
申请号：US10597755
申请日：2005-02-09
申请人： Vasant Jadhav , Shawn Zinnen
发明人： Vasant Jadhav , Shawn Zinnen
IPC分类号： C07H21/04
CPC分类号： C12N15/1131 , A61K38/00 , C12N15/1136 , C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/3519 , C12N2310/51 , C12N2310/53
摘要： The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to multifunctional short interfering nucleic acid (multifunctional siNA) molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism via RNA interference (RNAi). The bifunctional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA 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基于核酸的试剂，包括在治疗，兽医，农业，诊断，靶标验证和基因组发现应用中的用途。具体地，本发明涉及通过RNA干扰（RNAi）调节生物系统例如细胞，组织或生物体中的一种或多种基因的表达的多功能短干扰核酸（多功能siNA）分子。本发明的双功能短干扰核酸（多功能siNA）分子可以靶向单个靶核酸分子中的多于一个核酸序列区域，或者靶向不同靶核酸分子中核酸序列的区域。自身多功能siNA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。

6. 发明申请

US20050209180A1 RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA) 审中-公开
标题翻译：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：US20050209180A1
公开(公告)日：2005-09-22
申请号：US10942560
申请日：2004-09-15
申请人： Vasant Jadhav , Karl Kossen , Shawn Zinnen , Narendra Vaish , James McSwiggen
发明人： Vasant Jadhav , Karl Kossen , Shawn Zinnen , Narendra Vaish , James McSwiggen
IPC分类号： A61K48/00 , C07H21/02
摘要： This invention relates to compounds, compositions, and methods useful for modulating HCV gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of HCV gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features 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 and methods used to modulate the expression of HCV genes.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节HCV基因表达的化合物，组合物和方法。本发明还涉及可用于通过使用小核酸分子调节参与通过RNA干扰（RNAi）的HCV基因表达和/或活性的途径的其它基因的表达和活性的化合物，组合物和方法。特别地，本发明的特征在于小核酸分子，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））分子和用于调节HCV基因表达的方法。

7. 发明申请

US20060281175A1 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) 审中-公开
公开(公告)号：US20060281175A1
公开(公告)日：2006-12-14
申请号：US11502876
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： C12N5/06 , 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.

8. 发明申请

US20060217335A1 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) 审中-公开
公开(公告)号：US20060217335A1
公开(公告)日：2006-09-28
申请号：US11358807
申请日：2006-02-21
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 , C07H21/00 , 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.

9. 发明申请

US20050020525A1 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) 审中-公开
标题翻译：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：US20050020525A1
公开(公告)日：2005-01-27
申请号：US10757803
申请日：2004-01-14
申请人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimen Wang , Tonqian Chen , Narendra Vaish
发明人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimen Wang , Tonqian Chen , Narendra Vaish
IPC分类号： A61K38/00 , A61K47/48 , C12N15/113 , C12N15/87 , A61K48/00 , C07H21/02
CPC分类号： A61K49/0008 , A61K38/00 , A61K47/544 , A61K47/549 , A61K47/551 , A61K47/554 , A61K47/59 , A61K47/60 , A61K47/64 , A61K47/645 , C12N15/113 , C12N15/87 , C12N2310/111 , C12N2310/14 , C12N2310/141 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , 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。

10. 发明申请

US20140329768A1 VITAMIN B6 DERIVATIVES OF NUCLEOTIDES, ACYCLONUCLEOTIDES AND ACYCLONUCLEOSIDE PHOSPHONATES 有权
公开(公告)号：US20140329768A1
公开(公告)日：2014-11-06
申请号：US14236178
申请日：2012-08-01
申请人： Alexander Karpeisky , Shawn Zinnen , Murali Urlam , Vladimir Y. Vvedensky , Andrei P. Guzaev
发明人： Alexander Karpeisky , Shawn Zinnen , Murali Urlam , Vladimir Y. Vvedensky , Andrei P. Guzaev
IPC分类号： C07H19/10
CPC分类号： A61K31/7068 , C07D213/65 , C07D213/76 , C07H19/10 , C07H19/12 , C07H19/19 , C07H19/20 , C07H19/23
摘要： Provided herein are compounds, compositions, and methods of using them to treat infections, neoplastic disease, inflammatory disease, and pain. Such compounds are nucleotides, acyclonucleotides, and ANP phosphonates conjugated with forms and/or moieties of Vitamin B6 for delivery past the cell membrane and into the cell.

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