School of Nano-Tech and Nano-Bionics, University of Science and Technology of China (USTC), Hefei 230026, P. R. China.
CAS Key Laboratory for Nano-Bio Interface, Suzhou Institute of Nano-Tech and Nano-Bionics (SINANO), Chinese Academy of Science, Suzhou 215123, P. R. China.
J Mater Chem B. 2023 Jun 21;11(24):5251-5271. doi: 10.1039/d2tb02610d.
CRISPR/Cas systems are novel gene editing tools with tremendous capacity and accuracy for gene editing and hold great potential for therapeutic genetic manipulation. However, the lack of safe and efficient delivery methods for CRISPR/Cas and its guide RNA hinders their wide adoption for therapeutic applications. To this end, there is an increasing demand for safe, efficient, precise, and non-pathogenic delivery approaches, both and . With the convergence of nanotechnology and biomedicine, functional nanocomposites have demonstrated unparalleled sophistication to overcome the limits of CRISPR/Cas delivery. The tunability of the physicochemical properties of nanocomposites makes it very easy to conjugate them with different functional substances. The combinatorial application of diverse functional materials in the form of nanocomposites has shown excellent properties for CRISPR/Cas delivery at the target site with therapeutic potential. The recent highlights of selective organ targeting and phase I clinical trials for gene manipulation by CRISPR/Cas after delivery through LNPs are at the brink of making it to routine clinical practice. Here we summarize the recent advances in delivering CRISPR/Cas systems through nanocomposites for targeted delivery and therapeutic genome editing.
CRISPR/Cas 系统是一种新型的基因编辑工具，具有强大的基因编辑能力和准确性，在治疗性基因操作方面具有巨大的潜力。然而，CRISPR/Cas 及其向导 RNA 的安全有效的递送方法的缺乏阻碍了它们在治疗应用中的广泛采用。为此，人们对安全、高效、精确和非致病性的递送方法的需求越来越大，包括体内和体外。随着纳米技术和生物医学的融合，功能纳米复合材料表现出无与伦比的复杂性，克服了 CRISPR/Cas 递送的限制。纳米复合材料的物理化学性质的可调节性使得很容易将它们与不同的功能物质结合。不同功能材料以纳米复合材料的形式组合应用，在靶向部位具有出色的 CRISPR/Cas 递送性能和治疗潜力。最近，通过 LNPs 递送 CRISPR/Cas 进行基因操作的选择性器官靶向和 I 期临床试验的亮点即将成为常规临床实践。在这里，我们总结了通过纳米复合材料靶向递送和治疗性基因组编辑来递送 CRISPR/Cas 系统的最新进展。
