4-amino-2-[3-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-2,6-dioxopyrimidin-1-yl]butanoic acid

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - 嘧啶核苷
• 英文名称: 4-amino-2-[3-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-2,6-dioxopyrimidin-1-yl]butanoic acid
• 化学式: C₁₃H₁₉N₃O₈
• 分子量: 345.31
• InChiKey: LQWLXODDLJGJEZ-JANFQQFMSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -5.4
• 重原子数：
  24
• 可旋转键数：
  6
• 环数：
  2.0
• sp3杂化的碳原子比例：
  0.62
• 拓扑面积：
  174
• 氢给体数：
  5
• 氢受体数：
  9

文献信息

• Gene editing methods and compositions for eliminating risk of JC virus activation and PML (progressive multifocal leukoencephalopathy) during immunosuppressive therapy
  申请人：Excision BioTherapeutics, Inc.

  公开号：US10279014B2

  公开（公告）日：2019-05-07

  A method of eliminating the risk of JCV activation in a subject undergoing immunosuppressive therapy, by administering an effective amount of a gene editing composition directed toward at least one target sequence in the JCV genome, cleaving the target sequence in the JCV genome, disrupting the JCV genome, eliminating the JCV infection, eliminating the risk of JCV activation, and treating the subject with an immunosuppressive therapy. A pharmaceutical composition including at least one isolated nucleic acid sequence encoding a CRISPR-associated endonuclease and at least one gRNA having a spacer sequence complementary to a target sequence in a JCV DNA, the isolated nucleic acid sequences being included in at least one expression vector. Pharmaceutical compositions including at least one isolated nucleic acid sequence encoding at least one TALEN, at least one ZFN, and gene editing composition of C2c1, C2c3, TevCas9, Archaea Cas9, CasY.1-CasY.6, CasX, or argonaute protein, which target at least one nucleotide sequence of the JCV genome.

  一种消除接受免疫抑制治疗的受试者的JCV激活风险的方法，通过给药有效量的针对JCV基因组中至少一个靶序列的基因编辑组合物，裂解JCV基因组中的靶序列，破坏JCV基因组，消除JCV感染，消除JCV激活风险，并用免疫抑制疗法治疗受试者。一种药物组合物，包括至少一种编码 CRISPR 相关内切酶的分离核酸序列和至少一种具有与 JCV DNA 中靶序列互补的间隔序列的 gRNA，所述分离核酸序列包含在至少一种表达载体中。药物组合物，包括编码至少一种TALEN、至少一种ZFN和C2c1、C2c3、TevCas9、Archaea Cas9、CasY.1-CasY.6、CasX或argonaute蛋白的基因编辑组合物的至少一种分离核酸序列，其靶向JCV基因组的至少一种核苷酸序列。
• RNA GUIDED ERADICATION OF HUMAN JC VIRUS AND OTHER POLYOMAVIRUSES
  申请人：Temple University Of The Commonwealth

  公开号：EP3212795A1

  公开（公告）日：2017-09-06
• GENE EDITING METHODS AND COMPOSITIONS FOR ELIMINATING RISK OF JC VIRUS ACTIVATION AND PML (PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY) DURING IMMUNOSUPPRESIVE THERAPY
  申请人：Excision Biotherapeutics, Inc.

  公开号：EP3387001A2

  公开（公告）日：2018-10-17
• GENE EDITING METHODS AND COMPOSITIONS FOR ELIMINATING RISK OF JC VIRUS ACTIVATION AND PML (PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY) DURING IMMUNOSUPPRESSIVE THERAPY
  申请人：Excision BioTherapeutics, Inc.

  公开号：US20180140682A1

  公开（公告）日：2018-05-24

  A method of eliminating the risk of JCV activation in a subject undergoing immunosuppressive therapy, by administering an effective amount of a gene editing composition directed toward at least one target sequence in the JCV genome, cleaving the target sequence in the JCV genome, disrupting the JCV genome, eliminating the JCV infection, eliminating the risk of JCV activation, and treating the subject with an immunosuppressive therapy. A pharmaceutical composition including at least one isolated nucleic acid sequence encoding a CRISPR-associated endonuclease and at least one gRNA having a spacer sequence complementary to a target sequence in a JCV DNA, the isolated nucleic acid sequences being included in at least one expression vector. Pharmaceutical compositions including at least one isolated nucleic acid sequence encoding at least one TALEN, at least one ZFN, and gene editing composition of C2c1, C2c3, TevCas9, Archaea Cas9, CasY.1-CasY.6, CasX, or argonaute protein, which target at least one nucleotide sequence of the JCV genome.
• [EN] RNA GUIDED ERADICATION OF HUMAN JC VIRUS AND OTHER POLYOMAVIRUSES<br/>[FR] ÉRADICATION GUIDÉE PAR L'ARN DU VIRUS JC HUMAIN ET D'AUTRES POLYOMAVIRUS
  申请人：UNIV TEMPLE

  公开号：WO2016070070A1

  公开（公告）日：2016-05-06

  The present invention includes methods and compositions for elimination of polyomaviruses, such as John Cunningham Virus (JVC), from host cells, and the treatment of polyomavirus related diseases, such as progressive multifocal leukoencephalopathy (PML). The compositions include isolated nucleic acid sequences comprising a CRISPR-associated endonuclease and a guide RNA, wherein the guide RNA is complementary to a target sequence in a polyomavirus.