5-(1-丙炔)-2-o-甲基尿苷 | 179817-95-9

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - 嘧啶核苷
• 中文名称: 5-(1-丙炔)-2-o-甲基尿苷
• 英文名称: 5-(1-Propynyl)-2'-o-methyluridine
• 英文别名: 1-[(2R,3R,4R,5R)-4-hydroxy-5-(hydroxymethyl)-3-methoxyoxolan-2-yl]-5-prop-1-ynylpyrimidine-2,4-dione
• CAS: 179817-95-9
• 化学式: C₁₃H₁₆N₂O₆
• 分子量: 296.28
• InChiKey: KPJZKNCZUWDUIF-DNRKLUKYSA-N

物化性质

• 密度：
  1.48±0.1 g/cm3(Predicted)

计算性质

• 辛醇/水分配系数(LogP)：
  -1
• 重原子数：
  21
• 可旋转键数：
  4
• 环数：
  2.0
• sp3杂化的碳原子比例：
  0.54
• 拓扑面积：
  108
• 氢给体数：
  3
• 氢受体数：
  6

文献信息

• [EN] POLYNUCLEOTIDES ENCODING LIPOPROTEIN LIPASE FOR THE TREATMENT OF HYPERLIPIDEMIA<br/>[FR] POLYNUCLÉOTIDES CODANT POUR LA LIPOPROTÉINE LIPASE DESTINÉS AU TRAITEMENT DE L'HYPERLIPIDÉMIE
  申请人：MODERNATX INC

  公开号：WO2017201333A1

  公开（公告）日：2017-11-23

  The invention relates to mRNA therapy for the treatment of hyperlipidemia. mRNAs for use in the invention, when administered in vivo, encode human lipoprotein lipase (LPL), isoforms thereof, functional fragments thereof, and fusion proteins comprising LPL. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto, mRNA therapeaies of the invention increase and/or restore deficient levels of LPL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of triglycerides associated with deficient LPL activity in subjects.

  该发明涉及mRNA疗法用于治疗高脂血症。该发明中使用的mRNA，在体内给予时，编码人类脂蛋白脂肪酶（LPL）、其异构体、其功能性片段以及包含LPL的融合蛋白。该发明中的mRNA最好被封装在脂质纳米颗粒（LNPs）中，以实现对受试者的细胞和/或组织的高效传递，当给予治疗时，该发明中的mRNA疗法增加和/或恢复受试者中LPL表达和/或活性的不足水平。该发明中的mRNA疗法进一步降低与受试者中LPL活性不足相关的甘油三酯水平。
• PEG LIPIDS AND USES THEREOF
  申请人：Moderna TX, Inc.

  公开号：US20220047518A1

  公开（公告）日：2022-02-17

  The present disclosure in part provides compounds (i.e., PEG lipids) which are useful in pharmaceutical compositions, cosmetic compositions, and drug delivery systems, e.g, for use in lipid nanoparticle (LNP) formulations. The present disclosure also provides LNP formulations comprising PEG lipids described herein, and methods of using the same. For example, the LNPs provided herein are useful for the delivery of an agent (e.g, therapeutic agent) to a subject. The PEG lipids and LNPs provided herein, in certain embodiments, exhibit increased PEG shedding compared to existing PEG lipids and LNP formulations.

  本公开部分提供了化合物（即PEG脂质），这些化合物在制药组合物、化妆品组合物和药物递送系统中非常有用，例如用于脂质纳米粒（LNP）配方。本公开还提供了包含此处所述PEG脂质的LNP配方以及使用它们的方法。例如，此处提供的LNP对于将药物（例如治疗药物）递送到主体中非常有用。在某些实施例中，此处提供的PEG脂质和LNP表现出比现有的PEG脂质和LNP配方更高的PEG脱落。
• MODIFIED NUCLEIC ACID MOLECULES AND USES THEREOF
  申请人：MODERNA THERAPEUTICS, INC.

  公开号：US20150307542A1

  公开（公告）日：2015-10-29

  The present disclosure provides modified nucleosides, nucleotides, and nucleic acids, and methods of using them.

  本公开提供了修改的核苷酸，核苷酸和核酸，以及使用它们的方法。
• Novel nucleoside and oligonucleotide analogues
  申请人：SANKYO COMPANY, LIMITED

  公开号：US20020147332A1

  公开（公告）日：2002-10-10

  A compound of the formula (1): 1 wherein R 1 and R 2 are the same or different and represent a hydrogen atom, a hydroxyl protecting group, a phosphate group, or —P(R 3 )R 4 , wherein R 3 and R 4 are the same or different and represent a hydroxyl group, an amino group, an alkoxy group having from 1 to 4 carbon atoms, a cyanoalkoxy group having from 1 to 5 carbon atoms or an amino group substituted by an alkyl group having from 1 to 4 carbon atoms; A represents an alkylene group having from 1 to 4 carbon atoms and B represents a purin-9-yl group, a 2-oxo-pyrimidin-1-yl group, a substituted purin-9-yl group or a substituted 2-oxo-pyrimidin-1-yl group having a substituent &agr; selected from the group consisting of a hydroxyl group which may be protected, an alkoxy group having from 1 to 4 carbon atoms, a mercapto group which may be protected, an alkylthio group having from 1 to 4 carbon atoms, an alkoxy group having from 1 to 4 carbon atoms, an amino group which may be protected, a mono- or di-alkylamino group which may be substituted by an alkyl group having from 1 to 4 carbon atoms, an alkyl group having from 1 to 4 carbon atoms and a halogen atom; or a salt thereof.

  化合物的式子（1）：1其中R1和R2相同或不同，表示氢原子，羟基保护基，磷酸基或-P（R3）R4，其中R3和R4相同或不同，表示羟基，氨基，具有1至4个碳原子的烷氧基，具有1至5个碳原子的氰基烷氧基或被1至4个碳原子的烷基取代的氨基; A表示具有1至4个碳原子的烷基，B表示嘌呤-9-基，2-氧代嘧啶-1-基，取代的嘌呤-9-基或取代的2-氧代嘧啶-1-基，具有α取代基，所述α取代基选自羟基（可能被保护），具有1至4个碳原子的烷氧基，巯基（可能被保护），具有1至4个碳原子的烷硫基，具有1至4个碳原子的烷氧基，氨基（可能被保护），可以被1至4个碳原子的烷基取代的单烷基或双烷基氨基，具有1至4个碳原子的烷基和卤原子; 或其盐。
• ALTERNATIVE NUCLEIC ACID MOLECULES AND USES THEREOF
  申请人：Moderna Therapeutics, Inc.

  公开号：EP3053585A1

  公开（公告）日：2016-08-10

  The present disclosure provides alternative nucleosides, nucleotides, and nucleic acids, and methods of using them.

  本公开提供了可供选择的核苷、核苷酸和核酸以及使用它们的方法。