α-氨基酸 N-羧酸酐（NCA）衍生的用于核酸递送的合成多肽。
α-Amino acid N-carboxyanhydride (NCA)-derived synthetic polypeptides for nucleic acids delivery.
机构信息
Jiangsu Key Laboratory for Carbon-Based Functional Materials and Devices, Institute of Functional Nano & Soft Materials (FUNSOM), Collaborative Innovation Center of Suzhou Nano Science and Technology, Soochow University, Suzhou 215123, China.
Jiangsu Key Laboratory for Carbon-Based Functional Materials and Devices, Institute of Functional Nano & Soft Materials (FUNSOM), Collaborative Innovation Center of Suzhou Nano Science and Technology, Soochow University, Suzhou 215123, China.
出版信息
Adv Drug Deliv Rev. 2021 Apr;171:139-163. doi: 10.1016/j.addr.2020.12.007. Epub 2020 Dec 14.
In recent years, gene therapy has come into the spotlight for the prevention and treatment of a wide range of diseases. Polypeptides have been widely used in mediating nucleic acid delivery, due to their versatilities in chemical structures, desired biodegradability, and low cytotoxicity. Chemistry plays an essential role in the development of innovative polypeptides to address the challenges of producing efficient and safe gene vectors. In this Review, we mainly focused on the latest chemical advances in the design and preparation of polypeptide-based nucleic acid delivery vehicles. We first discussed the synthetic approach of polypeptides via ring-opening polymerization (ROP) of N-carboxyanhydrides (NCAs), and introduced the various types of polypeptide-based gene delivery systems. The extracellular and intracellular barriers against nucleic acid delivery were then outlined, followed by detailed review on the recent advances in polypeptide-based delivery systems that can overcome these barriers to enable in vitro and in vivo gene transfection. Finally, we concluded this review with perspectives in this field.
近年来，基因治疗因其在广泛疾病的预防和治疗方面的潜力而备受关注。由于其化学结构的多样性、所需的生物降解性和低细胞毒性，多肽被广泛用于介导核酸递送。化学在开发创新多肽以应对生产高效和安全基因载体的挑战方面发挥着重要作用。在这篇综述中，我们主要集中讨论了设计和制备基于多肽的核酸递送载体方面的最新化学进展。我们首先讨论了通过 N-羧酸酐（NCA）开环聚合（ROP）合成多肽的方法，并介绍了各种类型的基于多肽的基因递送系统。然后概述了核酸递送面临的细胞外和细胞内障碍，接着详细综述了能够克服这些障碍以实现体外和体内基因转染的基于多肽的递送系统的最新进展。最后，我们对该领域的前景进行了总结。
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