3a(2)-Uridylic acid, 2a(2)-O-methyl- | 54687-08-0

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - 核糖核苷3'-磷酸盐
• 英文名称: 3a(2)-Uridylic acid, 2a(2)-O-methyl-
• 英文别名: [(2R,3R,4R,5R)-5-(2,4-dioxopyrimidin-1-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-yl] dihydrogen phosphate
• CAS: 54687-08-0
• 化学式: C₁₀H₁₅N₂O₉P
• 分子量: 338.21
• InChiKey: NPRNBAPBGVERRF-ZOQUXTDFSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -3.1
• 重原子数：
  22
• 可旋转键数：
  5
• 环数：
  2.0
• sp3杂化的碳原子比例：
  0.6
• 拓扑面积：
  155
• 氢给体数：
  4
• 氢受体数：
  9

文献信息

• [EN] RNAI AGENTS FOR INHIBITING EXPRESSION OF BETA-ENAC, COMPOSITIONS THEREOF, AND METHODS OF USE<br/>[FR] AGENTS D'ARNI POUR INHIBER L'EXPRESSION DE BETA-ENAC, LEURS COMPOSITIONS ET PROCÉDÉS D'UTILISATION
  申请人：ARROWHEAD PHARMACEUTICALS INC

  公开号：WO2021086995A1

  公开（公告）日：2021-05-06

  Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a beta-ENaC (SCNN1B) gene. The beta-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a beta-ENaC gene. Pharmaceutical compositions that include one or more beta-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described beta-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of beta-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including chronic obstructive pulmonary disease (COPD).

  描述了RNAi药剂、包含RNAi药剂的组合物，以及抑制β-ENaC（SCNN1B）基因的方法。本文披露的β-ENaC RNAi药剂和RNAi药剂结合物抑制了β-ENaC基因的表达。还描述了包含一个或多个β-ENaC RNAi药剂的药物组合物，可选地与一个或多个额外治疗药物一起使用。将所述的β-ENaC RNAi药剂传递至上皮细胞，如体内的肺上皮细胞，可实现抑制β-ENaC基因表达和减少ENaC活性，这可以为受试者，包括人类受试者，提供治疗益处，用于治疗包括慢性阻塞性肺疾病（COPD）在内的各种疾病。
• Lipid Formulated Compositions and Methods for Inhibiting Expression of Serum Amyloid A Gene
  申请人：de Fougerolles Antonin

  公开号：US20110263684A1

  公开（公告）日：2011-10-27

  The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Serum Amyloid A (SAA) gene, and methods of using the dsRNA to inhibit expression of SAA.

  这项发明涉及一种针对血清淀粉样蛋白A（SAA）基因的双链核糖核酸（dsRNA），以及使用该dsRNA抑制SAA表达的方法。
• Compositions and Methods for Inhibiting Expression of Transthyretin
  申请人：Sah Dinah Wen-Yee

  公开号：US20100120893A1

  公开（公告）日：2010-05-13

  The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.

  该发明涉及一种靶向前甲状腺素转运蛋白（TTR）基因的双链核糖核酸（dsRNA），以及使用该dsRNA来抑制TTR表达的方法。
• Compositions And Methods For Increasing Cellular Uptake Of RNAi Via SID-1
  申请人：Hinkle Gregory

  公开号：US20120016006A1

  公开（公告）日：2012-01-19

  The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Systemic RNA Interference Defective-1 (SID-I) gene, and methods of using the dsRNA to inhibit expression of SID-1.

  该发明涉及一种靶向系统RNA干扰缺陷-1（SID-1）基因的双链核糖核酸（dsRNA），以及使用dsRNA抑制SID-1表达的方法。
• SERPINA1 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：EP3412774A1

  公开（公告）日：2018-12-12

  The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.

  该发明涉及RNAi药剂，例如靶向Serpina1基因的双链RNAi药剂，以及使用这种RNAi药剂抑制Serpina1表达的方法，以及治疗患有Serpina1相关疾病（如肝脏疾病）的受试者的方法。