3,4-Bis(3,7,11,15-tetramethylhexadecoxy)butyl methanesulfonate

• 分子结构分类: 有机化合物 - 脂质和类脂质分子 - 异戊烯醇脂质
• 英文名称: 3,4-Bis(3,7,11,15-tetramethylhexadecoxy)butyl methanesulfonate
• 英文别名: 3,4-bis(3,7,11,15-tetramethylhexadecoxy)butyl methanesulfonate
• 化学式: C₄₅H₉₂O₅S
• 分子量: 745.3
• InChiKey: ZAIQPCMIEQBJFB-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  17.7
• 重原子数：
  51
• 可旋转键数：
  37
• 环数：
  0.0
• sp3杂化的碳原子比例：
  1.0
• 拓扑面积：
  70.2
• 氢给体数：
  0
• 氢受体数：
  5

文献信息

• COMPOSITIONS AND METHODS FOR SILENCING EBOLA VIRUS GENE EXPRESSION
  申请人：Protiva Biotherapeutics, Inc.

  公开号：US20150111945A1

  公开（公告）日：2015-04-23

  The present invention provides compositions comprising therapeutic nucleic acids (e.g., interfering RNA such as siRNA) that target Ebola virus (EBOV) gene expression and methods of using such compositions to silence EBOV gene expression. More particularly, the invention provides unmodified and chemically modified interfering RNA which silence EBOV gene expression and methods of use thereof, e.g., for preventing or treating EBOV infections caused by one or more EBOV species such as Zaire EBOV. The invention also provides serum-stable nucleic acid-lipid particles comprising one or more interfering RNA molecules, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. Methods of silencing EBOV gene expression by administering one or more interfering RNA molecules to a mammalian subject are also provided.

  本发明提供了包含治疗性核酸（例如干扰RNA，如siRNA）的组合物，其靶向埃博拉病毒（EBOV）基因表达，并使用这些组合物来沉默EBOV基因表达的方法。更具体地，本发明提供了未修饰和化学修饰的干扰RNA，其沉默EBOV基因表达的方法以及使用这些方法，例如预防或治疗由一个或多个EBOV物种（例如扎伊尔EBOV）引起的EBOV感染。本发明还提供了血清稳定的核酸-脂质颗粒，包括一个或多个干扰RNA分子，一个阳离子脂质和一个非阳离子脂质，其还可以包括一种共轭脂质，抑制颗粒的聚集。本发明还提供了通过向哺乳动物受试者注射一个或多个干扰RNA分子来沉默EBOV基因表达的方法。
• CATIONIC LIPIDS AND METHODS FOR THE DELIVERY OF THERAPEUTIC AGENTS
  申请人：Protiva Biotherapeutics, Inc.

  公开号：US20140134260A1

  公开（公告）日：2014-05-15

  The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid in vivo. The compositions of the present invention are highly potent, thereby allowing effective know-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.

  本发明提供了一种用于向细胞传递治疗剂的组合物和方法。特别地，这些包括新颖的阳离子脂质和核酸-脂质颗粒，它们提供了核酸的高效封装和体内高效传递。本发明的组合物非常有效，因此可以在相对较低的剂量下有效地知道一个特定的靶蛋白。此外，本发明的组合物和方法比以前在艺术上已知的组合物和方法毒性更小，提供了更大的治疗指数。
• US8716464B2
  申请人：——

  公开号：US8716464B2

  公开（公告）日：2014-05-06
• US9018187B2
  申请人：——

  公开号：US9018187B2

  公开（公告）日：2015-04-28
• US9187748B2
  申请人：——

  公开号：US9187748B2

  公开（公告）日：2015-11-17