Attia Noha, Mashal Mohamed, Puras Gustavo, Pedraz Jose Luis
Laboratory of Pharmaceutics, NanoBioCel Research Group, School of Pharmacy, University of the Basque Country (UPV/EHU), Paseo de la Universidad 7, 01006 Vitoria-Gasteiz, Spain.
Department of Basic Sciences, The American University of Antigua-College of Medicine, Coolidge 1451, Antigua and Barbuda.
Pharmaceutics. 2021 Jun 7;13(6):843. doi: 10.3390/pharmaceutics13060843.
The cell-based approach in gene therapy arises as a promising strategy to provide safe, targeted, and efficient gene delivery. Owing to their unique features, as homing and tumor-tropism, mesenchymal stem cells (MSCs) have recently been introduced as an encouraging vehicle in gene therapy. Nevertheless, non-viral transfer of nucleic acids into MSCs remains limited due to various factors related to the main stakeholders of the process (e.g., nucleic acids, carriers, or cells). In this review, we have summarized the main types of nucleic acids used to transfect MSCs, the pros and cons, and applications of each. Then, we have emphasized on the most efficient lipid-based carriers for nucleic acids to MSCs, their main features, and some of their applications. While a myriad of studies have demonstrated the therapeutic potential for engineered MSCs therapy in various illnesses, optimization for clinical use is an ongoing challenge. On the way of improvement, genetically modified MSCs have been combined with various novel techniques and tools (e.g., exosomes, spheroids, 3D-Bioprinting, etc.,) aiming for more efficient and safe applications in biomedicine.
基因治疗中基于细胞的方法作为一种有前景的策略出现，可提供安全、靶向且高效的基因递送。由于间充质干细胞（MSCs）具有归巢和肿瘤趋向性等独特特性，最近它们被引入作为基因治疗中一种令人鼓舞的载体。然而，由于与该过程的主要参与因素（如核酸、载体或细胞）相关的各种因素，核酸向MSCs的非病毒转移仍然有限。在这篇综述中，我们总结了用于转染MSCs的主要核酸类型、各自的优缺点及应用。然后，我们重点介绍了用于向MSCs递送核酸的最有效的脂质基载体、它们的主要特性及其一些应用。虽然大量研究已经证明了工程化MSCs治疗在各种疾病中的治疗潜力，但针对临床应用进行优化仍是一项持续的挑战。在改进的道路上，基因修饰的MSCs已与各种新技术和工具（如外泌体、球体、3D生物打印等）相结合，旨在在生物医学中实现更高效和安全的应用。
