通过纳米尺度的化物伊米达酸框架实现的CRISPR/Cas9基因组编辑机器的内体逃生和传递
Shahad K Alsaiari1, Sachin Patil1, Mram Alyami1
1Smart Hybrid Materials (SHMs) Laboratory, Advanced Membranes and Porous Materials Center, King Abdullah University of Science and Technology (KAUST) , Thuwal 23955-6900, Saudi Arabia.
Journal of the American Chemical Society
|December 23, 2017
概括
研究人员开发了纳米尺度的热利性意达框架 (ZIF) 来封装CRISPR/Cas9基因编辑工具. 这种新型的输送系统克服了尺寸和充电方面的挑战, 增强了遗传疾病的治疗潜力.
科学领域:
- 生物技术
- 纳米技术
- 基因编辑
背景情况:
- CRISPR/Cas9是一个强大的基因组编辑平台,
- 由于CRISPR/Cas9组件 (Cas9蛋白和sgRNA) 的大小和高电荷,它们的输送具有挑战性.
- 现有的输送方法在效率和生物相容性方面存在局限性.
研究的目的:
- 为CRISPR/Cas9组件开发一种新的传递系统.
- 克服与CRISPR/Cas9相关的交付挑战.
- 评估新输送系统的效率和生物相容性.
主要方法:
- 在纳米尺度的热利性意达框架 (ZIF) 中封装CRISPR/Cas9.
- 使用ZIF-8进行封装,形成CC-ZIF.
- 评估负载效率,内体逃生和基因淘汰效率.
主要成果:
- 在ZIF中实现了17%的CRISPR/Cas9加载效率.
- 证明了因质子化伊米达部分而增强的内体逃生.
- 在使用CC- ZIF的4天内成功降低了37%的绿色光蛋白基因表达.
- 核实了纳米级CC-ZIF的生物相容性和可扩展性.
结论:
- 纳米尺度的热利性伊米达框架为CRISPR/Cas9提供了有效的解决方案.
- 该CC-ZIF系统显示了高负载能力和可控的Cas9和sgRNA代码传递.
- 这种方法为CRISPR/Cas9基因编辑应用提供了一个有前途的,生物兼容和可扩展的平台.
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