2-[2-(2-Hydroxyethoxy)ethoxy]ethyl 4-methylpiperazine-1-carboxylate

• 分子结构分类: 有机化合物 - 有机氧化合物
• 英文名称: 2-[2-(2-Hydroxyethoxy)ethoxy]ethyl 4-methylpiperazine-1-carboxylate
• 英文别名: 2-[2-(2-hydroxyethoxy)ethoxy]ethyl 4-methylpiperazine-1-carboxylate
• 化学式: C₁₂H₂₄N₂O₅
• 分子量: 276.33
• InChiKey: CGCRFXNXVNRQIX-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -1
• 重原子数：
  19
• 可旋转键数：
  9
• 环数：
  1.0
• sp3杂化的碳原子比例：
  0.92
• 拓扑面积：
  71.5
• 氢给体数：
  1
• 氢受体数：
  6

文献信息

• ANTIBACTERIAL ANTISENSE OLIGONUCLEOTIDE AND METHOD
  申请人：Sarepta Therapeutics, Inc.

  公开号：US20140213737A1

  公开（公告）日：2014-07-31

  A method for enhancing, by at least 10 fold, the antibacterial activity of an antisense oligonucleotide composed of morpholino subunits linked by phosphorus-containing intersubunit linkages. The method includes one or both of: conjugating an arginine-rich carrier to a 3′ or 5′ end of the oligonucleotide and modifying the oligonucleotide to contain 20%-50% intersubunit linkages that are positively charged at physiological pH. Also disclosed is an antisense oligonucleotide having enhanced antibacterial activity by virtue of one or both modifications.

  一种增强抗菌活性的反义寡核苷酸方法，其由连接磷含有的亚单位的Morpholino亚单位组成，能够至少增强10倍。该方法包括以下一种或两种：将富含精氨酸的载体与寡核苷酸的3'端或5'端结合，以及修改寡核苷酸，使其在生理pH下含有20％-50％的亚单位链接呈正电荷。此外，还公开了一种通过一种或两种修改具有增强抗菌活性的反义寡核苷酸。
• Exon skipping oligomer conjugates for muscular dystrophy
  申请人：Sarepta Therapeutics, Inc.

  公开号：US10888578B2

  公开（公告）日：2021-01-12

  Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.

  本研究描述了与人类肌营养不良症基因中选定的目标位点互补的反义寡聚体共轭物，以诱导外显子 51 跳越。
• Processes for preparing phosphorodiamidate morpholino oligomers
  申请人：SAREPTA THERAPEUTICS, INC.

  公开号：US10947533B2

  公开（公告）日：2021-03-16

  Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomer synthesis while maintaining overall yield and purity of a synthesized oligomer.

  本文提供了制备低聚物（如吗啉低聚物）的工艺。本文所述的合成工艺有利于扩大低聚物的合成规模，同时保持合成低聚物的总体产量和纯度。
• EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
  申请人：Sarepta Therapeutics, Inc.

  公开号：EP3554554B1

  公开（公告）日：2022-09-07
• [EN] EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY<br/>[FR] OLIGOMÈRES À SAUTS D'EXONS ASSOCIÉS À LA DYSTROPHIE MUSCULAIRE
  申请人：SAREPTA THERAPEUTICS INC

  公开号：WO2020023688A1

  公开（公告）日：2020-01-30

  Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 2 skipping are described. In various aspects, antisense oligomers are described according to Formula (I): Formula (I) or a pharmaceutically acceptable salt thereof, wherein T, Nu, n, and R100 are defined herein.