5'-O-Hydroxyethyladenosine | 23643-41-6

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - 嘌呤核苷
• 英文名称: 5'-O-Hydroxyethyladenosine
• 英文别名: 5'-O-(2-Hydroxyaethyl)adenosin；5-O-(β-hydroxy-ethyl)-adenosin；O^5'-(2-hydroxy-ethyl)-adenosine；(2R,3R,4S,5R)-2-(6-aminopurin-9-yl)-5-(2-hydroxyethoxymethyl)oxolane-3,4-diol
• CAS: 23643-41-6
• 化学式: C₁₂H₁₇N₅O₅
• 分子量: 311.297
• InChiKey: ASRAXMUIGSJRDK-WOUKDFQISA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -2.6
• 重原子数：
  22
• 可旋转键数：
  5
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.58
• 拓扑面积：
  149
• 氢给体数：
  4
• 氢受体数：
  9

文献信息

• PCSK9 iRNA compositions and methods of use thereof
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US10125369B2

  公开（公告）日：2018-11-13

  The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit expression of PCSK9 and methods of treating subjects having a lipid disorder, such as a hyperlipidemia.

  本发明涉及靶向 PCSK9 基因的 RNAi 制剂，例如双链 RNAi 制剂，以及使用这种 RNAi 制剂抑制 PCSK9 表达的方法和治疗血脂紊乱（如高脂血症）患者的方法。
• Patatin-like phospholipase domain containing 3 (PNPLA3) iRNA compositions and methods of use thereof
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US10231988B2

  公开（公告）日：2019-03-19

  The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such RNAi agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.

  本发明涉及靶向Patatin-Like Phospholipase Domain Containing 3 (PNPLA3)基因的RNAi制剂，例如双链RNAi制剂，以及使用这种RNAi制剂抑制PNPLA3基因表达的方法和治疗患有非酒精性脂肪肝（NAFLD）和/或PNPLA3相关疾病的受试者的方法。
• TMPRSS6 iRNA compositions and methods of use thereof
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US10246713B2

  公开（公告）日：2019-04-02

  The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.

  本发明涉及靶向TMPRSS6基因的RNAi制剂，例如双链RNAi制剂，以及使用这种RNAi制剂抑制TMPRSS6表达的方法和治疗患有TMPRSS6相关疾病（例如铁超载相关疾病，如β地中海贫血或血色病）的受试者的方法。
• Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US10407679B2

  公开（公告）日：2019-09-10

  The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.

  本发明涉及靶向脂蛋白C3（APOC3）基因的RNAi制剂，如双链RNAi制剂，以及使用这种RNAi制剂抑制APOC3表达的方法和治疗APOC3相关疾病（如高甘油三酯血症）患者的方法。
• Compositions and methods for inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) gene expression
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US10478500B2

  公开（公告）日：2019-11-19

  The invention relates to RNAi agents, e.g. double-stranded RNAi agents, targeting the HAO1 gene, and method of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1. Described herein are double-stranded RNAi agents which inhibit the expression of a HA01 gene in a cell, such as a cell within a subject, e.g., a mammal, such as a human having a HAO1 associated disorder, and uses of such double-stranded RNAi agents. In certain aspects of the invention, substantially all of the nucleotides of an iRNA of the invention are modified.

  本发明涉及靶向HAO1基因的RNAi制剂，例如双链RNAi制剂，以及使用这种RNAi制剂抑制HAO1表达的方法和治疗患有PH1等疾病的受试者的方法。本文描述了抑制细胞中HA01基因表达的双链RNAi制剂，例如受试者（例如哺乳动物，例如患有HAO1相关疾病的人类）体内的细胞，以及这种双链RNAi制剂的用途。在本发明的某些方面，本发明的 iRNA 的核苷酸基本上全部被修饰。