生物灵感的时空管理向RNA疗法
Yutian Ma1, Shiyao Li2, Xin Lin3
1Division of Pharmacoengineering and Molecular Pharmaceutics, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, United States.
ACS nano
|December 13, 2023
概括
由于其负电荷,RNA传递面临着挑战. 本综述涵盖了用于增强RNA治疗的病毒,纳米粒子和水凝系统,重点关注释放机制和未来的临床转化.
科学领域:
- 生物医学工程 生物医学工程
- 药物运输 药物运输 药物运输
- 分子治疗学分子治疗学
背景情况:
- 基于核糖核酸 (RNA) 的疗法在疾病干预方面表现有前途,FDA批准的mRNA疫苗和siRNA药物就是例子.
- 在有效地将负电荷RNA传递到治疗应用的目标站点方面,仍然存在重大挑战.
研究的目的:
- 审查当前的病毒,纳米粒子和基于水凝的RNA传递系统.
- 讨论由环境刺激触发的RNA加载/释放机制.
- 总结RNA疗法交付的时间和空间方面,并确定未来的研究机会.
主要方法:
- 关于病毒载体,纳米粒子和基于水凝的RNA输送平台的综合文献综述.
- 分析受光,热,pH和酶影响的RNA加载和释放机制.
- 在输送系统中的物理和化学相互作用的评估.
主要成果:
- 概述各种RNA输送策略,包括修饰,结合体和多复合体.
- 刺激响应RNA释放动力学和准能力的详细描述.
- 关于RNA输送系统开发当前挑战和未来前景的总结.
结论:
- 先进的传递系统对于克服RNA固有的传递挑战至关重要.
- 刺激响应平台为向治疗提供精确的RNA释放控制.
- 需要进一步的研究来将这些RNA输送系统转化为临床实践.
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