Guangzhou Municipal and Guangdong Provincial Key Laboratory of Molecular Target & Clinical Pharmacology, the NMPA and State Key Laboratory of Respiratory Disease, School of Pharmaceutical Sciences, Guangzhou Medical University, Guangzhou 511436, China.
ACS Macro Lett. 2024 Oct 15;13(10):1251-1257. doi: 10.1021/acsmacrolett.4c00405. Epub 2024 Sep 11.
The prevailing design philosophy for polymeric vectors delivering siRNA is rooted in the post-transcriptional gene silencing (PTGS) mechanism. Yet, the transcriptional gene silencing (TGS) mechanism offers a potentially more durable silencing effect, which necessitates efficient siRNA delivery into the nucleus. However, it remains a challenge for the polymeric vectors to efficiently deliver siRNA into the nucleus. We have explored guanidinylated cyclic synthetic polypeptides (GCSPs) to enhance the nuclear delivery of siRNA, but an increased cytotoxicity and difficulty in producing the GCSPs on a large scale limit their utility. Herein, we simply prepare PEGylated guanidinylated linear synthetic polypeptides (PGLSPs) exhibiting improved membrane penetration, direct siRNA transport to the nucleus, reduced toxicity, high cellular uptake, and mitigation of protein corona formation. The PEGylation can effectively balance the vector's nuclear delivery capacity with other critical aspects of performances for siRNA delivery. Therefore, the PGLSPs hold promise as TGS-based delivery vectors, offering potential for future therapeutic applications.
聚合物载体传递 siRNA 的主流设计理念源于转录后基因沉默(PTGS)机制。然而,转录基因沉默(TGS)机制提供了一种潜在更持久的沉默效果,这需要将 siRNA 有效递送到细胞核内。然而,对于聚合物载体来说,将 siRNA 有效递送到细胞核内仍然是一个挑战。我们已经探索了胍基化环状合成多肽(GCSPs)来增强 siRNA 的核内递送,但是增加的细胞毒性和在大规模生产 GCSPs 方面的困难限制了它们的应用。在这里,我们简单地制备了聚乙二醇化胍基化线性合成多肽(PGLSPs),其表现出改善的膜穿透性、直接将 siRNA 转运到细胞核、降低毒性、高细胞摄取和减轻蛋白质冠形成的特性。PEG 化可以有效地平衡载体的核内递送能力与 siRNA 递送的其他关键性能方面。因此,PGLSP 有望成为基于 TGS 的递药载体,为未来的治疗应用提供了潜力。