Lin Meng, Wang Xueyan
Department of Pharmacy, West China Hospital, Sichuan University, Chengdu 610041, China.
Pharmaceutics. 2023 Dec 30;16(1):62. doi: 10.3390/pharmaceutics16010062.
Over the last decade, the clustered, regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has become the most promising gene editing tool and is broadly utilized to manipulate the gene for disease treatment, especially for cancer, which involves multiple genetic alterations. Typically, CRISPR/Cas9 machinery is delivered in one of three forms: DNA, mRNA, or ribonucleoprotein. However, the lack of efficient delivery systems for these macromolecules confined the clinical breakthrough of this technique. Therefore, a variety of nanomaterials have been fabricated to improve the stability and delivery efficiency of the CRISPR/Cas9 system. In this context, the natural biopolymer-based carrier is a particularly promising platform for CRISPR/Cas9 delivery due to its great stability, low toxicity, excellent biocompatibility, and biodegradability. Here, we focus on the advances of natural biopolymer-based materials for CRISPR/Cas9 delivery in the cancer field and discuss the challenges for their clinical translation.
在过去十年中，成簇规律间隔短回文重复序列（CRISPR）/CRISPR相关蛋白9（Cas9）系统已成为最具前景的基因编辑工具，并被广泛用于操纵基因以进行疾病治疗，尤其是针对涉及多种基因改变的癌症。通常，CRISPR/Cas9机制以三种形式之一递送：DNA、mRNA或核糖核蛋白。然而，这些大分子缺乏高效的递送系统限制了该技术的临床突破。因此，人们制备了多种纳米材料以提高CRISPR/Cas9系统的稳定性和递送效率。在此背景下，基于天然生物聚合物的载体因其高稳定性、低毒性、出色的生物相容性和生物可降解性，成为CRISPR/Cas9递送特别有前景的平台。在这里，我们重点介绍基于天然生物聚合物的材料在癌症领域用于CRISPR/Cas9递送的进展，并讨论其临床转化面临的挑战。
