智能纳米治疗策略用于交付CRISPR系统
Chao Chen1, Wu Zhong2, Shiyu Du1
1Jiangsu Collaborative Innovation Center of Chinese Medicinal Resources Industrialization, School of Medicine & Holistic Integrative Medicine, Nanjing University of Chinese Medicine, Nanjing 210023, China.
Acta pharmaceutica Sinica. B
|July 10, 2023
概括
基因编辑CRISPR提供了精确的生物医学研究进步. 本综述探讨了在临床环境中安全有效的CRISPR-Cas9基因疗法应用的智能纳米粒子传递系统.
科学领域:
- 生物医学工程 生物医学工程
- 分子生物学分子生物学
- 纳米技术 纳米技术
背景情况:
- 克里斯普尔基因编辑技术提供了高效率,精度和成本效益,彻底改变了生物医学研究.
- 在CRISPR传递方面的进步对于将基因编辑转化为临床治疗至关重要.
研究的目的:
- 审查CRISPR传递策略及其在基因治疗中的翻译潜力.
- 在体内分析CRISPR交付和系统局限性的障碍.
- 专注于对刺激有反应的纳米载体,用于智能CRISPR传递.
主要方法:
- 审查CRISPR传递系统的治疗应用.
- 分析在体内使用CRISPR技术的挑战和局限性.
- 纳米载体介导的CRISPR-Cas9传递策略的总结.
主要成果:
- 智能纳米粒子显示了CRISPR系统交付的巨大潜力.
- 目前正在开发各种刺激响应的纳米载体,用于有针对性的CRISPR传递.
- 新的基因组编辑器正在探索纳米治疗载体介导基因疗法.
结论:
- 对刺激有反应的纳米载体代表了安全有效的CRISPR传递的有希望的策略.
- 纳米载体系统的进一步开发对于推进基于CRISPR的基因疗法至关重要.
- 基因组编辑在优化纳米载体的临床环境中具有显著的未来前景.
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