刺激响应性核酸输送系统在基因治疗中的进步和挑战
Meng Lin1,2, Xianrong Qi1
1Beijing Key Laboratory of Molecular Pharmaceutics and New Drug Delivery System, School of Pharmaceutical Sciences, Peking University, 38 Xueyuan Road, Beijing 100191, China.
Pharmaceutics
|May 27, 2023
概括
响应刺激的纳米载体为治疗疾病提供智能基因传递. 这些系统克服了核酸输送的障碍,在临床翻译方面面临挑战,需要进一步研究有效的基因治疗.
科学领域:
- 生物医学工程 生物医学工程
- 纳米技术纳米技术
- 基因治疗 基因治疗
背景情况:
- 基因疗法通过纠正基因,为癌症和神经系统疾病等疾病提供持续治疗.
- 对于基因疗法至关重要的核酸,由于不稳定性和薄弱的膜透性,面临着交付挑战.
- 响应刺激的传递系统正在出现,以克服这些障碍,使控制和向的核酸释放成为可能.
研究的目的:
- 审查用于基因传递的刺激响应纳米载体的原理.
- 为突出突出响应刺激的基因传递系统的进步.
- 讨论这些纳米载体的临床转化所面临的挑战和解决方案.
主要方法:
- 关于用于基因传递的刺激响应纳米载体的当前文献的综述.
- 在纳米载体设计中使用的各种刺激 (pH,氧化还原,酶,光,磁场,超声波) 的分析.
- 检查与基于纳米载体的基因疗法相关的临床前和临床挑战.
主要成果:
- 响应刺激的纳米载体可以智能地控制核酸的释放,并针对特定的网站.
- 已经开发了各种纳米载体系统,利用内源和外部刺激.
- 大多数系统仍然处于临床前阶段,需要解决诸如转染效率,安全性,制造和非目标效应等问题.
结论:
- 对刺激有反应的纳米载体对推进基因疗法有很大的前景.
- 解决当前的挑战对于成功的临床翻译至关重要.
- 进一步的研究将加速这些先进的交付系统的开发和应用.
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