1-tert-butyl-N-ethyl-N-methylpiperidin-4-amine

• 分子结构分类: 有机化合物 - 有机杂环化合物 - 哌啶类
• 英文名称: 1-tert-butyl-N-ethyl-N-methylpiperidin-4-amine
• 化学式: C₁₂H₂₆N₂
• 分子量: 198.35
• InChiKey: UFQZSXKYZIDZNJ-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  2.2
• 重原子数：
  14
• 可旋转键数：
  3
• 环数：
  1.0
• sp3杂化的碳原子比例：
  1.0
• 拓扑面积：
  6.5
• 氢给体数：
  0
• 氢受体数：
  2

文献信息

• Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
  申请人：Sarepta Therapeutics, Inc.

  公开号：US11060089B2

  公开（公告）日：2021-07-13

  The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.

  本公开涉及修饰的反义寡核苷酸。本文所述的核苷酸具有 10 至 40 个核碱基，包括与人α葡萄糖苷酶（GAA）基因前核糖核酸内含子 1 中靶区互补的靶向序列。与靶向序列相比，靶区包括至少一个额外的核碱基，其中至少一个额外的核碱基在靶向序列中没有互补的核碱基，并且至少一个额外的核碱基在靶区内部。
• ANTISENSE-INDUCED EXON2 INCLUSION IN ACID ALPHA-GLUCOSIDASE
  申请人：Sarepta Therapeutics, Inc.

  公开号：US20150197534A1

  公开（公告）日：2015-07-16

  The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD-II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha-glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene.
• ANTISENSE OLIGOMERS AND METHODS OF USING THE SAME FOR TREATING DISEASES ASSOCIATED WITH THE ACID ALPHA-GLUCOSIDASE GENE
  申请人：Sarepta Therapeutics, Inc.

  公开号：US20190284555A1

  公开（公告）日：2019-09-19

  The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.