1-(1,5-anhydro-2-deoxy-D-galactitol-2-yl)-4-(4-cyanophenoxy)methyl-1,2,3-triazole | 1196658-77-1

• 分子结构分类: 有机化合物 - 苯类化合物 - 苯酚醚
• 英文名称: 1-(1,5-anhydro-2-deoxy-D-galactitol-2-yl)-4-(4-cyanophenoxy)methyl-1,2,3-triazole
• 英文别名: 4-[[1-[(3S,4R,5R,6R)-4,5-dihydroxy-6-(hydroxymethyl)oxan-3-yl]triazol-4-yl]methoxy]benzonitrile
• CAS: 1196658-77-1
• 化学式: C₁₆H₁₈N₄O₅
• 分子量: 346.343
• InChiKey: KCMBHZQWDQBHEE-JJXSEGSLSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -0.8
• 重原子数：
  25
• 可旋转键数：
  5
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.44
• 拓扑面积：
  134
• 氢给体数：
  3
• 氢受体数：
  8

反应信息

• 作为产物：
  描述：

  1-(3,4,6-tri-O-acetyl-1,5-anhydro-2-deoxy-D-galactitol-2-yl)-4-(4-cyanophenoxy)methyl-1,2,3-triazole 在 水 、 三乙胺 作用下， 以 甲醇 为溶剂， 以81%的产率得到1-(1,5-anhydro-2-deoxy-D-galactitol-2-yl)-4-(4-cyanophenoxy)methyl-1,2,3-triazole
  参考文献：
  名称：

  Design, synthesis and evaluation of monovalent ligands for the asialoglycoprotein receptor (ASGP-R)

  摘要：

  A series of novel aryl-substituted triazolyl D-galactosamine derivatives was synthesized as ligands for the carbohydrate recognition domain of the major subunit H1 (H1-CRD) of the human asialoglycoprotein receptor (ASGP-R). The compounds were biologically evaluated with a newly developed competitive binding assay, surface plasmon resonance and by a competitive NMR binding experiment. With compound 1b, a new ligand with a twofold improved affinity to the best so far known D-GalNAc was identified. This small, drug-like ligand can be used as targeting device for drug delivery to hepatocytes. (C) 2009 Elsevier Ltd. All rights reserved.

  DOI：

  10.1016/j.bmc.2009.08.049

文献信息

• [EN] OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF<br/>[FR] COMPOSITIONS D'OLIGONUCLÉOTIDES ET PROCÉDÉS ASSOCIÉS
  申请人：WAVE LIFE SCIENCES LTD

  公开号：WO2019075357A1

  公开（公告）日：2019-04-18

  Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).

  本公开提供了用于改变剪接的技术，特别是用于增加剪接产物中外显子包含量的技术。在某些实施例中，本公开提供了SMN2寡核苷酸、组合物及其方法。在某些实施例中，本公开提供了立体控制的SMN2寡核苷酸组合物。在某些实施例中，所提供的寡核苷酸和组合物可以增加外显子7包含的SMN2剪接产物的水平和/或其基因产物的水平。在某些实施例中，本公开提供了用于治疗剪接相关疾病、疾病和疾病的方法。在某些实施例中，本公开提供了用于治疗SMN2相关疾病、疾病和疾病的方法，如脊髓性肌萎缩症（SMA）和肌萎缩侧索硬化（ALS）。
• OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
  申请人：VARGEESE Chandra

  公开号：US20200157545A1

  公开（公告）日：2020-05-21

  Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
• OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
  申请人：WAVE LIFE SCIENCES LTD.

  公开号：US20210228615A1

  公开（公告）日：2021-07-29

  Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).