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使用阴离子迷纳米管传递siRNA用于基因沉默

Uyen Ho^1,2, Mounir El-Bakkari^1,2, Aws Alshamsan^2,3

¹Department of Chemistry, University of Alberta, 11227 Saskatchewan Drive Edmonton, Alberta, T6G 2G2, Canada.

Biomaterials science

|September 21, 2023

概括

Розет纳米管 (RNTs) 有效地传递小干扰RNA (siRNA) 用于基因沉默. 这些DNA纳米结构保护siRNA免受降解,并增强细胞吸收,减少约70%的蛋白表达.

科学领域:

生物材料科学生物材料科学
纳米技术纳米技术
分子生物学分子生物学
基因治疗基因治疗

背景情况:

RNA干扰 (RNAi) 和基因沉默为遗传性疾病提供治疗潜力.
有效的传递策略对于降低毒性和提高RNAi的沉默效率至关重要.
罗塞特纳米管 (RNTs),自组装从关氨酸-氨酸DNA,是有希望的仿生纳米结构.

研究的目的:

研究生物活性RNTs用于小干扰RNA (siRNA) 传递和基因沉默的使用.
为了合成和表征素功能化RNTs (KnT) 以提高siRNA复杂化和细胞吸收.
评估KnT RNTs在传递siRNA和在癌症细胞系中实现基因沉默方面的效率.

主要方法:

使用固相合成合成十五种素功能化双GC基因 (KnT,n=1到15).
在生理条件下,KnT图案的自我组装成阴离子RNT.
在HCT116和A549细胞中通过KnT RNTs介导的siRNA复杂化和细胞内吸收的评估.
基因表达分析以量化目标stat3蛋白表达的减少.

主要成果:

从素功能化的GC基因中成功形成了阴离子RNT.
KnT RNTs表现出高效的siRNA复杂化,受电荷和RNT长度的影响.
带有较高电荷的KnT RNTs通过内细胞通路促进了较快的siRNA细胞吸收.
KnT RNTs保护siRNA免受血清降解,导致显著的stat3蛋白表达减少 (~70%).

结论:

素功能化迷纳米管 (KnT RNTs) 代表了siRNA输送的有效和仿生平台.
KnT RNTs的阴离子性质和自我组装特性提高了siRNA吸收和基因沉默效率.
这种方法对开发更安全,更有效的基因沉默疗法来治疗遗传性疾病充满希望.

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