1-(9-Adenyl)-1'-(phosphoryloxymethyl)-diaethylenglykol | 4348-56-5

• 分子结构分类: 有机化合物 - 有机杂环化合物 - 咪唑并嘧啶类
• 英文名称: 1-(9-Adenyl)-1'-(phosphoryloxymethyl)-diaethylenglykol
• 英文别名: phosphoric acid mono-{2-[1-(6-amino-purin-9-yl)-2-hydroxy-ethoxy]-3-hydroxy-propyl} ester；[(2S)-2-[(1R)-1-(6-aminopurin-9-yl)-2-hydroxyethoxy]-3-hydroxypropyl] dihydrogen phosphate
• CAS: 4348-56-5
• 化学式: C₁₀H₁₆N₅O₇P
• 分子量: 349.24
• InChiKey: LJUZKMNCGRSCCT-NKWVEPMBSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -3.2
• 重原子数：
  23
• 可旋转键数：
  8
• 环数：
  2.0
• sp3杂化的碳原子比例：
  0.5
• 拓扑面积：
  186
• 氢给体数：
  5
• 氢受体数：
  11

文献信息

• Messenger UNA molecules and uses thereof
  申请人：Arcturus Therapeutics, Inc.

  公开号：US10815463B2

  公开（公告）日：2020-10-27

  This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.

  本发明提供了一系列可翻译的信使 UNA（mUNA）分子。这些 mUNA 分子可在体外和体内翻译，以提供活性多肽或蛋白质，或提供免疫制剂或疫苗成分。mUNA 分子可用作活性剂，在细胞或受试者体内表达活性多肽或蛋白质。除其他外，mUNA 分子还可用于治疗罕见疾病。
• Compositions and agents against hepatitis B virus and uses thereof
  申请人：Arcturus Therapeutics, Inc.

  公开号：US10961535B2

  公开（公告）日：2021-03-30

  This invention encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting Hepatitis B virus in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers, and the compounds are targeted to a sequence of an HBV genome.

  本发明包括在医学治疗方法中有用的化合物和组合物，一般来说，用于抑制受试者体内的乙型肝炎病毒。化合物具有第一链和第二链，每条链的长度为 19-29 个单体，单体包括 UNA 单体和核酸单体，化合物靶向 HBV 基因组序列。
• Therapeutics for glycogen storage disease type III
  申请人：Ultragenyx Pharmaceutical Inc.

  公开号：US11377643B2

  公开（公告）日：2022-07-05

  This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL), or a fragment thereof having AGL activity. The polynucleotide and oligomer molecules are expressible to provide the human AGL or a fragment thereof having AGL activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with reduced activity of amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL) in a subject.

  本发明提供了一系列可翻译的多核苷酸和寡聚体分子，用于表达人淀粉-α-1，6-葡萄糖苷酶、4-α-葡聚糖转移酶（AGL）或其具有 AGL 活性的片段。多核苷酸和寡聚物分子可表达出具有 AGL 活性的人 AGL 或其片段。这些分子可用作活性制剂，在细胞或人体中表达活性多肽或蛋白质。这些制剂可用于改善、预防、推迟发病或治疗与受试者体内淀粉-α-1，6-葡萄糖苷酶、4-α-葡聚糖转移酶（AGL）活性降低有关的疾病或病症的方法。
• MESSENGER UNA MOLECULES AND USES THEREOF
  申请人：Arcturus Therapeutics, Inc.

  公开号：US20180051262A1

  公开（公告）日：2018-02-22

  This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.
• THERAPEUTIC UNA OLIGOMERS AND USES THEREOF
  申请人：Arcturus Therapeutics, Inc.

  公开号：US20180105816A1

  公开（公告）日：2018-04-19

  This invention provides UNA oligomers for regulating the expression of a target gene. The UNA oligomers contain UNA monomer linkers, and may contain one or more nucleotides modified with a 2′-O-methyl group, one or more nucleotides modified with a 2′-deoxy-2′-fluoro group, and one or more phosphorothioate or chiral phosphorothioate intermonomer linkages. UNA oligomers can be used as active agents for preventing or treating disease.