[hydroxy-[[(2R,3R,4R,5R)-3-hydroxy-4-methoxy-5-(6-oxo-1H-purin-9-yl)oxolan-2-yl]methoxy]phosphoryl] phosphono hydrogen phosphate

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - 嘌呤核苷酸
• 英文名称: [hydroxy-[[(2R,3R,4R,5R)-3-hydroxy-4-methoxy-5-(6-oxo-1H-purin-9-yl)oxolan-2-yl]methoxy]phosphoryl] phosphono hydrogen phosphate
• 化学式: C₁₁H₁₇N₄O₁₄P₃
• 分子量: 522.19
• InChiKey: ODAFAEFSKRENSW-IOSLPCCCSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -5.4
• 重原子数：
  32
• 可旋转键数：
  9
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.55
• 拓扑面积：
  258
• 氢给体数：
  6
• 氢受体数：
  15

文献信息

• ANTISENSE OLIGONUCLEOTIDES AND USES THEREOF
  申请人：Fundació Centre de Regulació Genòmica

  公开号：EP3543340A1

  公开（公告）日：2019-09-25

  Disclosed herein are novel compounds comprising antisense oligonucleotides that regulate the splicing of NUMB. In particular, an antisense oligonucleotide for reducing inclusion of NUMB exon 9 in a population of mature NUMB transcripts is provided. The antisense oligonucleotide comprises a sequence of at least 7 nucleotides that is complementary to a target region within exon 9 of a NUMB transcript. Pharmaceutical compositions comprising the antisense oligonucleotide and methods of treating a proliferative disease using the compounds or compositions of the invention are also described.

  本文公开了由反义寡核苷酸组成的新型化合物，这些反义寡核苷酸可调节 NUMB 的剪接。特别是提供了一种反义寡核苷酸，用于减少成熟 NUMB 转录本群体中 NUMB 外显子 9 的包含。反义寡核苷酸包括至少 7 个核苷酸的序列，该序列与 NUMB 转录本第 9 外显子内的目标区域互补。还描述了包含反义寡核苷酸的药物组合物和使用本发明化合物或组合物治疗增殖性疾病的方法。
• Isolation of target nucleic acids
  申请人：GENETICS RESEARCH, LLC

  公开号：US11142788B2

  公开（公告）日：2021-10-12

  The invention provides methods of isolating a target nucleic acid in a sample. A primer is hybridized to the target. A polymerase and modified nucleotide resistant to nuclease degradation are used to extend the primer to create a modified polynucleotide. The sample is exposed to a nuclease, thereby isolating the modified polynucleotide. Optionally, the target nucleic acid may be further protected by binding a protein in a sequence specific manner to one end of the target nucleic acid to create a protected target nucleic acid resistant to nuclease degradation. Thus, after exposing the sample to a nuclease, the modified polynucleotide and protected target nucleic acid are isolated.

  本发明提供了在样品中分离目标核酸的方法。引物与目标核酸杂交。使用聚合酶和耐核酸酶降解的修饰核苷酸扩展引物，形成修饰多核苷酸。将样本暴露于核酸酶，从而分离出修饰的多核苷酸。可以选择将蛋白质以序列特异的方式与目标核酸的一端结合，进一步保护目标核酸，以产生抗核酸酶降解的受保护目标核酸。这样，将样品暴露于核酸酶后，就能分离出修饰的多核苷酸和受保护的靶核酸。
• Nucleic acid conjugates and uses thereof
  申请人：Translate Bio MA, Inc.

  公开号：US11253601B2

  公开（公告）日：2022-02-22

  Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targeting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof; and kits for the preparation of conjugates described herein.

  本发明提供了包含靶向分子（如糖、叶酸盐和细胞穿透肽）的共轭物，可用于改善向细胞递送制剂（如核酸，如寡核苷酸或 mRNA，或其他制剂）。本发明提供了包含靶向分子的共轭物和化合物、制备方法以及用于制备它们的中间体。在另一方面，本发明提供了包含靶向分子的共轭物和化合物的制剂（如药物组合物）。本发明还提供了向细胞递送制剂（如核酸，如寡核苷酸或 mRNA）的方法，治疗和/或预防受试者疾病或病症的方法，以及调节细胞或受试者基因表达的方法。此外，本文还提供了包含共轭物或其制剂的试剂盒；以及制备本文所述共轭物的试剂盒。
• Modified Diguanylate Cyclase-Phosphodiesterase and Method for Enzymatic Production of Cyclic-diGMP
  申请人：NESBITT Natasha M.

  公开号：US20140170689A1

  公开（公告）日：2014-06-19

  The present invention provides a recombinant polypeptide comprising a first portion and a second portion, wherein the sequence of the first portion is fully identical to amino acids 1 to 248 of the sequence set forth as SEQ ID NO:1 and the sequence of the second portion is other than amino acids 249 to 511 of the sequence set forth as SEQ ID NO:1.
• COMPOSITIONS AND METHODS FOR MODULATING RNA
  申请人：RaNA Therapeutics, Inc.

  公开号：US20150050738A1

  公开（公告）日：2015-02-19

  Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the invention disclosed herein provide methods and compositions that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).