9-[(2R,3R,4S,5R)-4-hydroxy-5-(hydroxymethyl)-3-methoxyoxolan-2-yl]-1H-purin-6-one

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - 嘌呤核苷
• 英文名称: 9-[(2R,3R,4S,5R)-4-hydroxy-5-(hydroxymethyl)-3-methoxyoxolan-2-yl]-1H-purin-6-one
• 化学式: C₁₁H₁₄N₄O₅
• 分子量: 282.25
• InChiKey: HPHXOIULGYVAKW-GZCUOZMLSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -1.6
• 重原子数：
  20
• 可旋转键数：
  3
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.55
• 拓扑面积：
  118
• 氢给体数：
  3
• 氢受体数：
  6

文献信息

• COMPOSITIONS AND METHODS RELATED TO THERAPEUTIC CELL SYSTEMS EXPRESSING EXOGENOUS RNA
  申请人：RUBIUS THERAPEUTICS, INC.

  公开号：US20190161730A1

  公开（公告）日：2019-05-30

  The invention includes compositions and methods related to erythroid cells comprising exogenous RNA encoding a protein. The exogenous RNA can comprise a heterologous untranslated region comprising a regulatory element. Alternatively or in combination, the exogenous RNA can comprise chemical modifications.
• AMPLIFIABLE RNAS FOR THERAPEUTIC CELL SYSTEMS
  申请人：RUBIUS THERAPEUTICS, INC.

  公开号：US20190330591A1

  公开（公告）日：2019-10-31

  This disclosure provides, among other things, amplifiable nucleic acid constructs for expressing a gene of interest in a cell, e.g., an erythroid cell. The amplifiable nucleic acid construct may contain the gene of interest and an RNA-dependent RNA polymerase (RdRP)-responsive 5′ UTR, and may optionally further contain an RdRP-responsive 3′ UTR. RdRP may also be provided, e.g., on the same construct or a different construct.
• MICRORNA INHIBITORS FOR USE IN TREATING METABOLIC DISEASES
  申请人：Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheilt und Umwelt (GmbH)

  公开号：US20210254067A1

  公开（公告）日：2021-08-19

  The present invention relates to composition comprising an inhibitor of miR-379 or a portion or fragment thereof and an inhibitor of miR-541 or a portion or fragment thereof, and/or an inhibitor of the target site of miR-379 or a portion or fragment thereof and an inhibitor of the target site of miR-541 or a portion or fragment thereof, and/or a combination of an inhibitor of miR-379 or a portion or fragment thereof and an inhibitor of the target site of miR-541 or a portion or fragment thereof or a combination of an inhibitor of the target site of miR-379 or a portion or fragment thereof and an inhibitor of miR-541 or a portion or fragment thereof. The present invention also relates to the respective composition for use in treating or preventing a metabolic disease, a disease related to a metabolic disorder, and/or cancer.
• METHOD FOR DIAGNOSING DISEASES USING MULTIPLEX FLUORESCENCE AND SEQUENCING
  申请人：MultiplexDX, s.r.o.

  公开号：US20220002812A1

  公开（公告）日：2022-01-06

  The present invention relates to methods for diagnosing a disease by determining via multiplex fluorescence in situ hybridization (FISH) whether or not mRNA species and/or at least one miRNA species of disease-associated biomarkers ar present in a sample obtained from a subject, and by determining by multiplex sequencing whether or not said mRNA species of disease-associated biomarkers and/or said miRNA species of disease-associated biomarkers of step (a) are present in said sample. The present invention also relates to kits for performing the methods for diagnosis as described and provided herein as well as use of such kits for performing the methods for diagnosis as described and provided herein.
• Amplifiable RNAs for Therapeutic Cell Systems
  申请人：Rubius Therapeutics, Inc.

  公开号：US20220073875A1

  公开（公告）日：2022-03-10

  This disclosure provides, among other things, amplifiable nucleic acid constructs for expressing a gene of interest in a cell, e.g., an erythroid cell. The amplifiable nucleic acid construct may contain the gene of interest and an RNA-dependent RNA polymerase (RdRP)-responsive 5′ UTR, and may optionally further contain an RdRP-responsive 3′ UTR. RdRP may also be provided, e.g., on the same construct or a different construct.