2-Ethyl-4-methyl-6-phenylmorpholine

• 分子结构分类: 有机化合物 - 有机杂环化合物 - 恶嗪烷类
• 英文名称: 2-Ethyl-4-methyl-6-phenylmorpholine
• 英文别名: 2-ethyl-4-methyl-6-phenylmorpholine
• 化学式: C₁₃H₁₉NO
• 分子量: 205.3
• InChiKey: DYWXFAAERGVKKI-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  2.4
• 重原子数：
  15
• 可旋转键数：
  2
• 环数：
  2.0
• sp3杂化的碳原子比例：
  0.54
• 拓扑面积：
  12.5
• 氢给体数：
  0
• 氢受体数：
  2

文献信息

• MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
  申请人：Sarepta Therapeutics, Inc.

  公开号：EP3133160A1

  公开（公告）日：2017-02-22

  The present invention relates to an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping. The present invention further relates to a pharmaceutical composition comprising an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping.

  本发明涉及一种寡核苷酸，该寡核苷酸与诱导外显子跳越的人类肌营养不良素基因外显子中的靶区特异性杂交。本发明进一步涉及一种药物组合物，其中包含与诱导外显子跳越的人肌萎缩蛋白基因外显子中的靶区特异性杂交的寡核苷酸。
• dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
  申请人：Iversen Patrick L.

  公开号：US10017763B2

  公开（公告）日：2018-07-10

  Morpholino oligomers containing modified intersubunit linkages and/or terminal groups are provided for use within dsRNA molecules. The oligomers are oligonucleotide analogs containing predetermined sequences of base-pairing moieties. Also provided are such oligomers conjugated to peptide transporter moieties, where the transporters are preferably composed of arginine subunits, or arginine dimers, alternating with neutral amino acid subunits.

  Morpholino 寡聚物含有修饰的亚基间连接和/或末端基团，可用于 dsRNA 分子。这些寡聚物是含有预定碱基配对分子序列的寡核苷酸类似物。还提供了与肽转运体分子连接的此类寡聚体，其中转运体最好由精氨酸亚基或精氨酸二聚体组成，与中性氨基酸亚基交替使用。
• Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA
  申请人：Sarepta Therapeutics, Inc.

  公开号：US10398721B2

  公开（公告）日：2019-09-03

  Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.

  本研究提供了治疗类早衰症及相关疾病患者的方法，这些方法依靠 LMNA 靶向反义寡核苷酸来降低一种或多种编码早衰素的异常剪接 LMNA mRNA 异构体的表达。
• Induced exon inclusion in spinal muscle atrophy
  申请人：Sarepta Therapeutics, Inc.

  公开号：US10577605B2

  公开（公告）日：2020-03-03

  The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment for Spinal Muscle Atrophy (SMA). More particularly it relates to inducing inclusion of exon 7 to restore levels of Survival Motor Neuron (SMN) protein encoded by the Survival Motor Neuron (SMN) gene.

  本发明涉及使用反义化合物诱导外显子内含作为脊髓性肌肉萎缩症（SMA）的治疗方法。更具体地说，它涉及诱导外显子 7 的包含，以恢复由生存运动神经元 (SMN) 基因编码的生存运动神经元 (SMN) 蛋白的水平。
• DsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
  申请人：Sarepta Therapeutics, Inc.

  公开号：US11072793B2

  公开（公告）日：2021-07-27

  Morpholino oligomers containing modified intersubunit linkages and/or terminal groups are provided for use within dsRNA molecules. The oligomers are oligonucleotide analogs containing predetermined sequences of base-pairing moieties. Also provided are such oligomers conjugated to peptide transporter moieties, where the transporters are preferably composed of arginine subunits, or arginine dimers, alternating with neutral amino acid subunits.

  Morpholino 寡聚物含有修饰的亚基间连接和/或末端基团，可用于 dsRNA 分子。这些寡聚物是含有预定碱基配对分子序列的寡核苷酸类似物。还提供了与肽转运体分子连接的此类寡聚体，其中转运体最好由精氨酸亚基或精氨酸二聚体组成，与中性氨基酸亚基交替使用。