基于CRISPR的基因组编辑技术,称为成簇的规则间隔短回文重复(CRISPR),引发了人们对基因治疗的新兴趣。这种兴趣伴随着单向导RNA(sgRNA)的发展,这使得当与CRISPR组分一起使用时能够在靶向位点引入所需的遗传修饰。然而,CRISPR/Cas的有效交付仍然是一个挑战。成功的基因编辑依赖于能够有效地将CRISPR货物递送到目标位点的递送策略的开发。为了克服这个障碍,研究人员广泛探索非病毒,病毒,以及用于将CRISPR/Cas9和向导RNA(gRNA)靶向递送到细胞和组织中的物理方法。在这些方法中,脂质体提供了一种有希望的方法来增强CRISPR/Cas和gRNA的递送。脂质体促进内体逃逸和利用各种刺激,如光,pH值,超声,和环境线索,以提供货物释放的空间和时间控制。因此,基于CRISPR的系统与脂质体递送技术的组合能够在细胞和组织中实现精确和有效的遗传修饰。这种方法在基础研究中有许多应用,生物技术,和治疗干预措施。例如,它可用于纠正与遗传性疾病和其他疾病相关的基因突变,或修饰免疫细胞以增强其抗病能力。总之,基于脂质体的CRISPR基因组编辑为实现精确和有效的遗传修饰提供了有价值的工具。这篇综述讨论了进一步推进这一快速发展领域的未来方向和机会。
The CRISPR-based genome editing technology, known as clustered regularly interspaced short palindromic repeats (CRISPR), has sparked renewed interest in gene therapy. This interest is accompanied by the development of single-guide RNAs (sgRNAs), which enable the introduction of desired genetic modifications at the targeted site when used alongside the CRISPR components. However, the efficient delivery of CRISPR/Cas remains a challenge. Successful gene editing relies on the development of a delivery strategy that can effectively deliver the CRISPR cargo to the target site. To overcome this obstacle, researchers have extensively explored non-viral, viral, and physical methods for targeted delivery of CRISPR/Cas9 and a guide RNA (gRNA) into cells and tissues. Among those methods, liposomes offer a promising approach to enhance the delivery of CRISPR/Cas and gRNA. Liposomes facilitate endosomal escape and leverage various stimuli such as light, pH, ultrasound, and environmental cues to provide both spatial and temporal control of cargo release. Thus, the combination of the CRISPR-based system with liposome delivery technology enables precise and efficient genetic modifications in cells and tissues. This approach has numerous applications in basic research, biotechnology, and therapeutic interventions. For instance, it can be employed to correct genetic mutations associated with inherited diseases and other disorders or to modify immune cells to enhance their disease-fighting capabilities. In summary, liposome-based CRISPR genome editing provides a valuable tool for achieving precise and efficient genetic modifications. This review discusses future directions and opportunities to further advance this rapidly evolving field.