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德罗伊丁硫酸盐涂层的异质双重分子球形核酸

Toni Laine¹, Prasannakumar Deshpande², Ville Tähtinen¹

¹Department of Chemistry, University of Turku, 20500, Turku, Finland.

Chembiochem : a European journal of chemical biology

|November 15, 2024

概括

携带反意义寡核酸 (ASO) 的分子球形核酸 (MSNA) 有效地将Tau-ASO传递给神经元. 在MSNA上ondroitin硫酸盐的修改增强了细胞吸收和tau蛋白抑制在体外.

科学领域:

纳米技术和药物输送
神经科学和分子生物学

背景情况:

分子球状核酸 (MSNA) 是树突型纳米结构,具有作为寡核酸输送载体的潜力.
可以调节MSNA的辐射结构和外球合体,以影响传递特性.
反感性寡核酸 (ASO) 用于基因沉默,但它们的传递到细胞仍然是一个挑战.

研究的目的:

调查基于富勒的MSNA作为Tau-ASO的载体.
评估氏丁硫酸盐 (CS) 结合和装饰对MSNA介导的Tau-ASO的递送和活性的影响.
在体外评估这些新型MSNA-ASO合物的形成,稳定性,细胞吸收和Tau抑制活性.

主要方法:

[60]基于富勒的MSNA携带DNA和RNA序列的合成.
-ASO与不同硫化模式的里丁硫酸盐四糖 (CS) 的结合.
使用UV化,PAGE,DLS和SEC-MALS进行MSNA-ASO合物形成和稳定性的表征.
通过共聚焦显微镜进行细胞吸收的体外评估和通过海马神经元中的西部斑点通过Tau抑制活动.

主要成果:

异重组ASO-MSNAs成功形成并以稳定性为特征.
该MSNA载体和CS-moieties影响了tau-ASO的细胞吸收和tau抑制活动.
在MSNA上的CS装饰调节了Tau-ASO在海马神经元中的传递效率和生物活性.

结论:

基于富勒烯的MSNA是有效的杂交介导载体,可以携带反感性寡核酸.
氏丁硫酸盐的结合和装饰是一种可行的策略,可以增强MSNA介导的基因沉默.
这些发现凸显了定制MSNA-ASO合物的潜力,用于神经科学中的治疗应用.

关键词:

孔德罗二硫酸盐的使用方法药物运输药物运输药物运输异种双重的ASO是异种双重的分子球形核酸分子球形核酸纳米颗粒纳米颗粒

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