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治愈的障碍:克服病毒和非病毒基因疗法的细胞障碍

Steffen Honrath¹, Michael Burger¹, Jean-Christophe Leroux¹

¹ETH Zurich, Department of Chemistry and Applied Biosciences, Institute of Pharmaceutical Sciences, Vladimir-Prelog-Weg 3, 8093 Zurich, Switzerland.

International journal of pharmaceutics

|March 20, 2025

概括

基因传递面临生物障碍,但工程化病毒和非病毒载体显示出希望. 矢量设计的进步正在提高未来疗法的效率和安全性.

科学领域:

生物技术是生物技术.
分子生物学分子生物学
基因治疗基因治疗

背景情况:

基因传递对于治疗疾病至关重要,但面临着重大的生物障碍.
这些障碍包括影响治疗疗效的细胞外,细胞和细胞内障碍.

研究的目的:

审查基因传递中的细胞障碍.
用先进的矢量系统综合当前关于克服这些障碍的知识.

主要方法:

对病毒和非病毒基因传递载体的审查.
分析矢量工程的最新进展,包括可电离的脂质,可生物降解的聚合物和工程蛋白系统.

主要成果:

病毒载体提供高效率,但具有免疫性和载荷限制.
非病毒载体提供更安全的替代品,但与细胞内障碍和传染效率作斗争.
最近的工程工作已经部分解决了内体逃逸,毒性和传递效率.

结论:

克服生物障碍是推进基因传递系统的关键.
持续的载体工程对于开发更安全,更有效的基因疗法至关重要.
解决体内可翻译性,可扩展性和免疫性问题仍然至关重要.

关键词:

阻碍基因传递的障碍基因输送基因输送基因输送非病毒载体非病毒载体病毒载体病毒载体

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