PDF(Pubmed)
Abstract：
Gene manipulation of hematopoietic stem cells (HSCs) using the CRISPR/Cas system as a potent genome editing tool holds immense promise for addressing hematologic disorders. An essential hurdle in advancing this treatment lies in effectively delivering CRISPR/Cas to HSCs. While various delivery formats exist, Ribonucleoprotein complex (RNP) emerges as a particularly efficient option. RNP complexes offer enhanced gene editing capabilities, devoid of viral vectors, with rapid activity and minimized off-target effects. Nevertheless, novel delivery methods such as microfluidic-based techniques, filtroporation, nanoparticles, and cell-penetrating peptides are continually evolving. This study aims to provide a comprehensive review of these methods and the recent research on delivery approaches of RNP complexes to HSCs.
摘要：
使用CRISPR/Cas系统作为有效的基因组编辑工具对造血干细胞（HSC）进行基因操作，对于解决血液系统疾病具有巨大的希望。推进这种治疗的一个重要障碍在于有效地将CRISPR/Cas递送至HSC。虽然存在各种交付格式，核糖核蛋白复合物（RNP）是一种特别有效的选择。RNP复合物提供增强的基因编辑能力，没有病毒载体，具有快速的活动和最小化的脱靶效应。然而，新的递送方法，如基于微流体的技术，filtroporation,纳米粒子，和细胞穿透肽不断进化。本研究旨在对这些方法以及RNP复合物向HSC的递送方法的最新研究进行全面综述。
