School of Pharmacy, Hainan Medical University, Haikou 571199, China.
Department of Pediatrics, University Hospitals Rainbow Babies & Children's Hospital, Cleveland, OH 44106, USA.
Cells. 2024 Mar 24;13(7):568. doi: 10.3390/cells13070568.
Solid tumors, with their intricate cellular architecture and genetic heterogeneity, have long posed therapeutic challenges. The advent of the CRISPR genome editing system offers a promising, precise genetic intervention. However, the journey from bench to bedside is fraught with hurdles, chief among them being the efficient delivery of CRISPR components to tumor cells. Lipid nanoparticles (LNPs) have emerged as a potential solution. This biocompatible nanomaterial can encapsulate the CRISPR/Cas9 system, ensuring targeted delivery while mitigating off-target effects. Pre-clinical investigations underscore the efficacy of LNP-mediated CRISPR delivery, with marked disruption of oncogenic pathways and subsequent tumor regression. Overall, CRISPR/Cas9 technology, when combined with LNPs, presents a groundbreaking approach to cancer therapy, offering precision, efficacy, and potential solutions to current limitations. While further research and clinical testing are required, the future of personalized cancer treatment based on CRISPR/Cas9 holds immense promise.
实体肿瘤具有复杂的细胞结构和遗传异质性,长期以来一直是治疗的挑战。CRISPR 基因组编辑系统的出现提供了一种有前途的精确基因干预方法。然而,从实验室到临床应用的道路充满了障碍,其中主要的障碍是将 CRISPR 组件有效地递送到肿瘤细胞中。脂质纳米颗粒(LNPs)已成为一种潜在的解决方案。这种生物相容性纳米材料可以包裹 CRISPR/Cas9 系统,确保靶向递送的同时减轻脱靶效应。临床前研究强调了 LNP 介导的 CRISPR 递送的疗效,明显破坏了致癌途径,随后肿瘤消退。总的来说,CRISPR/Cas9 技术与 LNPs 结合,为癌症治疗提供了一种开创性的方法,具有精确性、疗效和对当前局限性的潜在解决方案。虽然还需要进一步的研究和临床测试,但基于 CRISPR/Cas9 的个性化癌症治疗的未来具有巨大的潜力。