siRNA纳米颗粒的配方,转染和增强的粘附 - 透到鼻子粘膜组织中
Leslie Vanessa Sanchez-Castillo1, Fabiola Guareschi2, Eleni Tsekoura1
1Department of Chemical and Materials Engineering, Faculty of Engineering, University of Alberta, 116 St & 85 Avenue, Edmonton, AB T6G 2G6, Canada.
三甲基基托 (TMC) 纳米颗粒有效地提供基因治疗剂,如用于EGFR基因沉默的小干扰RNA (siRNA). 这些纳米颗粒显示出有希望的粘膜粘合和粘膜透,用于非侵入性输送.
科学领域:
- 生物技术是生物技术.
- 纳米医学是一种纳米医学.
- 基因治疗 基因治疗
背景情况:
- 基因疗法对治疗疾病具有前景,但有效的传递系统至关重要.
- 小干扰RNA (siRNA) 为基因沉默提供了一个有针对性的方法.
- 为siRNA开发安全高效的输送载体是纳米医学的一个关键挑战.
研究的目的:
- 为了评估三甲基基托 (TMC) 纳米颗粒用于小干扰RNA (siRNA) 输送.
- 为了优化TMC纳米粒子配方的复杂化,释放和转化效率.
- 评估TMC纳米颗粒的粘膜粘合和粘膜透特性,以进行非侵入性输送.
主要方法:
- 在各种siRNA:TMC重量对重量比率下制备TMC纳米粒子.
- 结合效率的评估,释放特征 (用肝素),物理性质和细胞毒性.
- 在HeLa细胞中对EGFR敲击效率的评估.
- 探索添加剂 (德克斯特兰硫酸盐,三聚酸盐,氨酸) 以提高性能.
- 在子鼻腔粘膜上使用双光子显微镜进行粘结和粘膜透研究.
主要成果:
- 较高的TMC比率增强了siRNA结合,降低了药物释放率.
- 添加剂提高了纳米粒子的稳定性.
- 优化的配方表现出高细胞活力和显著的EGFR基因沉默.
- 与自由siRNA相比,TMC纳米颗粒显示出优异的粘膜粘合和粘膜透.
结论:
- TMC纳米粒子是siRNA传递的有效载体,可以实现显著的EGFR基因沉默.
- 配方优化和添加剂的使用提高了纳米粒子的性能.
- 由于其粘粘性和粘膜透性特性,TMC纳米颗粒显示出非侵入性基因疗法应用的潜力.
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