6-amino-1H-pyrimidin-2-one;1,7-dihydropurin-6-one

• 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: 6-amino-1H-pyrimidin-2-one;1,7-dihydropurin-6-one
• 化学式: C9H9N7O2
• 分子量: 247.21
• InChiKey: CRYRGPNRAULTHU-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -1.0
• 重原子数：
  18
• 可旋转键数：
  0
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.0
• 拓扑面积：
  138
• 氢给体数：
  4
• 氢受体数：
  4

文献信息

• [EN] OLIGONUCLEOTIDE THERAPY FOR WOLMAN DISEASE AND CHOLESTERYL ESTER STORAGE DISEASE<br/>[FR] THÉRAPIE OLIGONUCLÉOTIDIQUE POUR MALADIE DE WOLMAN ET MALADIE DE STOCKAGE DES ESTERS DU CHOLESTÉROL
  申请人：DEEP GENOMICS INCORPORATED

  公开号：WO2020237391A1

  公开（公告）日：2020-12-03

  The present disclosure provides antisense oligonucleotides, compositions, and methods that target a LIPA intron flanking exon 8, thereby modulating splicing of LIPA pre-mRNA to increase the level of LIPA mRNA molecules having exon 8, e.g., to provide a therapy for Wolman Disease or Cholesteryl Ester Storage Disease. The present disclosure provides an antisense oligonucleotide including a nucleobase sequence at least 70% complementary to a LIPA pre-mRNA target sequence in a 5'-flanking intron, a 3'-flanking intron, or a combination of exon 8 and the 5'-flanking or 3'-flanking intron.

  本公开提供了针对LIPA内含子环绕外显子8的反义寡核苷酸、组合物和方法，从而调节LIPA前mRNA的剪接，增加具有外显子8的LIPA mRNA分子的水平，例如，为Wolman病或胆固醇酯储存病提供治疗。本公开提供了一种包括至少70％与LIPA前mRNA靶序列在5'-环绕内含子、3'-环绕内含子或外显子8和5'-环绕或3'-环绕内含子的组合中互补的核碱序列的反义寡核苷酸。
• Compositions and methods of nucleic acid-targeting nucleic acids
  申请人：Caribou Biosciences, Inc.

  公开号：US10125361B2

  公开（公告）日：2018-11-13

  This disclosure provides for compositions and methods for the use of nucleic acid-targeting nucleic acids and complexes thereof.

  本公开提供了使用核酸靶向核酸及其复合物的组合物和方法。
• Synthetic RIG-I-like receptor agonists
  申请人：Checkmate Pharmaceuticals, Inc.

  公开号：US10907161B2

  公开（公告）日：2021-02-02

  The present disclosure relates to, inter alia, RNA molecules (e.g., RNA hairpin agonists) that bind to and agonize RIG-I-like receptors (RLRs), and to use of the molecules in methods for treating, or ameliorating one or more symptoms of, a disorder (e.g., cancer).

  本公开特别涉及与 RIG-I 样受体（RLR）结合并激动 RIG-I 样受体的 RNA 分子（如 RNA 发夹激动剂），以及将这些分子用于治疗或改善一种或多种疾病（如癌症）症状的方法。
• Method of target cleaving using CRISPR hybrid DNA/RNA polynucleotides
  申请人：Caribou Biosciences, Inc.

  公开号：US10988781B2

  公开（公告）日：2021-04-27

  The present disclosure provides DNA-guided CRISPR systems; polynucleotides comprising DNA, RNA and mixtures thereof for use with CRISPR systems; and methods of use involving such polynucleotides and DNA-guided CRISPR systems.

  本公开提供了 DNA 引导的 CRISPR 系统；包含 DNA、RNA 及其混合物的用于 CRISPR 系统的多核苷酸；以及涉及此类多核苷酸和 DNA 引导的 CRISPR 系统的使用方法。
• Compositions and methods for genomic editing by insertion of donor polynucleotides
  申请人：CRISPR THERAPEUTICS AG

  公开号：US11332760B2

  公开（公告）日：2022-05-17

  The present disclosure provides donor polynucleotides, genome editing systems, methods, pharmaceutical compositions, and kits which correct or induce a mutation that causes Glycogen Storage Disease 1a in a genomic DNA (gDNA) molecule in a cell. In some embodiments the present disclosure provides donor polynucleotides comprising two strands capable of correcting a mutation that causes Glycogen Storage Disease 1a.

  本公开提供了供体多核苷酸、基因组编辑系统、方法、药物组合物和试剂盒，它们可以纠正或诱导细胞中基因组DNA（gDNA）分子中导致糖原贮积症1a的突变。在一些实施方案中，本公开提供的供体多核苷酸包含两条链，能够纠正导致糖原贮积症1a的突变。