(2S)-1-[2-(2-hydroxyethoxy)ethoxy]-3-phenylmethoxypropan-2-ol

• 分子结构分类: 有机化合物 - 苯类化合物 - 苯和取代衍生物
• 英文名称: (2S)-1-[2-(2-hydroxyethoxy)ethoxy]-3-phenylmethoxypropan-2-ol
• 化学式: C₁₄H₂₂O₅
• 分子量: 270.32
• InChiKey: NBHGNLCQKXLUAJ-CQSZACIVSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  0
• 重原子数：
  19
• 可旋转键数：
  11
• 环数：
  1.0
• sp3杂化的碳原子比例：
  0.57
• 拓扑面积：
  68.2
• 氢给体数：
  2
• 氢受体数：
  5

文献信息

• THERAPEUTIC AGENT FOR GLYCOGEN STORAGE DISEASE TYPE IA
  申请人：DAIICHI SANKYO COMPANY, LIMITED

  公开号：US20200407394A1

  公开（公告）日：2020-12-31

  The present invention establishes a molecular therapy for glycogen storage disease type Ia. The present invention provides an oligonucleotide of 15-30 bases comprising a nucleotide sequence complementary to the cDNA of G6PC gene with c.648G>T mutation, wherein the oligonucleotide comprises a sequence complementary to a region comprising any site between the 82 nd to the 92 nd nucleotide from the 5′ end of exon 5 of the G6PC gene with c.648G>T mutation, a pharmacologically acceptable salt or solvate thereof. Also provided is a pharmaceutical drug comprising the oligonucleotide, a pharmacologically acceptable salt or solvate thereof (e.g., therapeutic drug for glycogen storage disease type Ia).