[5-(6-Aminopurin-9-yl)-4-hydroxy-2-(hydroxymethyl)oxolan-3-yl] [5-(2,4-dioxopyrimidin-1-yl)-4-hydroxy-3-phosphonooxyoxolan-2-yl]methyl hydrogen phosphate

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - （3'->5'）-二核苷酸和类似物
• 英文名称: [5-(6-Aminopurin-9-yl)-4-hydroxy-2-(hydroxymethyl)oxolan-3-yl] [5-(2,4-dioxopyrimidin-1-yl)-4-hydroxy-3-phosphonooxyoxolan-2-yl]methyl hydrogen phosphate
• 化学式: C₁₉H₂₅N₇O₁₅P₂
• 分子量: 653.4
• InChiKey: FZCSEXOMUJFOHQ-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -6.5
• 重原子数：
  43
• 可旋转键数：
  10
• 环数：
  5.0
• sp3杂化的碳原子比例：
  0.53
• 拓扑面积：
  321
• 氢给体数：
  8
• 氢受体数：
  19

文献信息

• METHOD FOR INCREASING FETAL HEMOGLOBIN EXPRESSION LEVEL
  申请人：EDIGENE INC.

  公开号：US20200384032A1

  公开（公告）日：2020-12-10

  Provided is a method for gene editing of an enhancer site of the BCL11A in hematopoietic stem cells. The genetically modified hematopoietic stem cells have the functions of normal cells, and can significantly increase the expression of fetal hemoglobin so as to be used in the treatment of β thalassemia and sickle cell anemia.