Poly(oxy-1,2-ethanediyl), I+/--hydro-I-[2-[[3-(2,5-dihydro-2,5-dioxo-1H-pyrrol-1-yl)-1-oxopropyl]amino]ethoxy]-, ether with 2,2-bis(hydroxymethyl)-1,3-propanediol (4:1) | 1349099-55-3

• 分子结构分类: 有机化合物 - 有机杂环化合物 - 吡咯烷类
• 英文名称: Poly(oxy-1,2-ethanediyl), I+/--hydro-I-[2-[[3-(2,5-dihydro-2,5-dioxo-1H-pyrrol-1-yl)-1-oxopropyl]amino]ethoxy]-, ether with 2,2-bis(hydroxymethyl)-1,3-propanediol (4:1)
• 英文别名: 3-(2,5-dioxopyrrol-1-yl)-N-[2-[2-[3-[2-[2-[3-(2,5-dioxopyrrol-1-yl)propanoylamino]ethoxy]ethoxy]-2,2-bis[2-[2-[3-(2,5-dioxopyrrol-1-yl)propanoylamino]ethoxy]ethoxymethyl]propoxy]ethoxy]ethyl]propanamide
• CAS: 1349099-55-3
• 化学式: C₄₉H₆₈N₈O₂₀
• 分子量: 1089.1
• InChiKey: CBXSONRSBAZBJH-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -6.8
• 重原子数：
  77
• 可旋转键数：
  44
• 环数：
  4.0
• sp3杂化的碳原子比例：
  0.59
• 拓扑面积：
  340
• 氢给体数：
  4
• 氢受体数：
  20

文献信息

• HYPERBRANCHED CATIONIC POLYMERS USEFUL AS NUCLEIC ACID DELIVERY VECTORS FOR TRANSFECTING
  申请人：University College Dublin

  公开号：EP3798250A1

  公开（公告）日：2021-03-31

  Hyperbranched cationic polymers are described. The polymers employ a 4-branching monomer resulting in an increase in the number of functional terminal groups due to the extra branching units, providing excellent transfection efficiency and cytocompatibility in different cell types, including aADSC, HeLa, Neu7 and RDEB keratinocytes, and delivering different genetic therapy approaches such GFP plasmid DNA and a ribonucleoprotein CRISP-Cas 9 complex for COL7A1 exon 80 skipping. In addition, the extra branching units of the polymer of the invention increases the positive charge on the polymer, which provides for improved endosomal escape within the cell. The 4-branching unit can be a diamine component, or a tetraacrylate component, although other 4-branching monomers may be employed such as for example any component with tetra acrylamide groups (i.e. 4-arm PEG acrylamide, 4-arm PEG maleimide), any component with tetra N-hydroxysuccinimide (NHS) groups (i.e. 4-arm PEG-succinimidyl carbonate NHS ester), any type of tetrathiol component (i.e. Pentaerythritol tetrakis(3-mercaptopropionate), 4-arm PEG-thiol, Tetra(2- mercaptoethyl)silane), and any tetraepoxy component (i.e. TetraGlycidyl methylenedianiline, Tetraglycidyl 1,1'-methylenebis(naphthalene-2,7-diol), Pentaerythritol tetraglycidyl ether, 4-arm peg epoxide).

  介绍了超支化阳离子聚合物。这种聚合物采用 4 支化单体，由于额外的支化单元，功能性末端基团的数量增加，在不同类型的细胞（包括 aADSC、HeLa、Neu7 和 RDEB 角质细胞）中具有优异的转染效率和细胞相容性，并可提供不同的基因治疗方法，如用于 COL7A1 外显子 80 跳接的 GFP 质粒 DNA 和核糖核蛋白 CRISP-Cas 9 复合物。此外，本发明聚合物的额外分支单元增加了聚合物的正电荷，从而改善了细胞内的内泌体逸散。4 支化单元可以是二胺组分或四丙烯酸酯组分，当然也可以使用其他 4 支化单体，例如具有四丙烯酰胺基团的任何组分（即 4 支 PEG 丙烯酰胺、4 支 PEG 马来酰亚胺）、具有四 N-羟基琥珀酰亚胺（NHS）基团的任何组分（即 4 支 PEG-琥珀酰亚胺）、具有四 N-羟基琥珀酰亚胺（NHS）基团的任何组分（即 4 支 PEG-琥珀酰亚胺）、具有四 N-羟基琥珀酰亚胺（NHS）基团的任何组分（即 4 支 PEG-琥珀酰亚胺）。4-臂 PEG-琥珀酰亚胺碳酸酯 NHS 酯）、任何类型的四硫醇成分（即季戊四醇四巯基（3-巯基丙酸酯）、4-臂 PEG-硫醇、四（2-巯基乙基）硅烷）以及任何四环氧成分（即四缩水甘油醚、四缩水甘油醚、四缩水甘油醚、四缩水甘油醚、四缩水甘油醚、四缩水甘油醚、四缩水甘油醚、四缩水甘油醚、四缩水甘油醚、四缩水甘油醚）。即四缩水甘油基亚甲基二苯胺、四缩水甘油基 1,1'-亚甲基双(萘-2,7-二醇)、季戊四醇四缩水甘油醚、4-臂聚环氧乙烷）。
• [EN] HYPERBRANCHED CATIONIC POLYMERS USEFUL AS NUCLEIC ACID DELIVERY VECTORS FOR TRANSFECTING CELLS<br/>[FR] POLYMÈRES CATIONIQUES HYPERRAMIFIÉS UTILES EN TANT QUE VECTEURS D'ADMINISTRATION D'ACIDES NUCLÉIQUES POUR LA TRANSFECTION DE CELLULES
  申请人：UNIV DUBLIN

  公开号：WO2021058491A1

  公开（公告）日：2021-04-01

  Hyperbranched cationic polymers are described. The polymers employ a 4-branching monomer resulting in an increase in the number of functional terminal groups due to the extra branching units, providing excellent transfection efficiency and cytocompatibility in different cell types, including aADSC, HeLa, Neu7 and RDEB keratinocytes, and delivering different genetic therapy approaches such GFP plasmid DNA and a ribonucleoprotein CRISP-Cas 9 complex for COL7A1 exon 80 skipping. In addition, the extra branching units of the polymer of the invention increases the positive charge on the polymer, which provides for improved endosomal escape within the cell. The 4-branching unit can be a diamine component, or a tetraacrylate component, although other 4-branching monomers may be employed such as for example any component with tetra acrylamide groups (i.e. 4-arm PEG acrylamide, 4-arm PEG maleimide), any component with tetra N- hydroxysuccinimide (NHS) groups (i.e. 4-arm PEG-succinimidyl carbonate NHS ester), any type of tetrathiol component (i.e. Pentaerythritol tetrakis(3-mercaptopropionate), 4-arm PEG-thiol, Tetra(2- mercaptoethyl)silane), and any tetraepoxy component (i.e. TetraGlycidyl methylenedianiline, Tetraglycidyl 1,1'-methylenebis(naphthalene-2,7-diol), Pentaerythritol tetraglycidyl ether, 4-arm peg epoxide).