Department of Metallurgical, Materials, and Biomedical Engineering, College of Engineering, University of Texas at El Paso, 500 W. University Ave., El Paso, Texas 79968, United States.
ACS Appl Bio Mater. 2024 Aug 19;7(8):5020-5036. doi: 10.1021/acsabm.3c00886. Epub 2024 Jan 30.
In medicine, it is desirable for clinicians to be able to restore function and imbue novel function into selected cells for therapy and disease prevention. Cells damaged by disease, injury, or aging could be programmed to restore normal or lost functions, such as retinal cells in inherited blindness and neuronal cells in Alzheimer's disease. Cells could also be genetically programmed with novel functions such as immune cells expressing synthetic chimeric antigen receptors for immunotherapy. Furthermore, knockdown or modification of risk factor proteins can mitigate disease development. Currently, nucleic acids are emerging as a versatile and potent therapeutic modality for achieving this cellular programming. In this review, we highlight the latest developments in nanobiomaterials-based nucleic acid therapeutics for cellular programming from a biomaterial design and delivery perspective and how to overcome barriers to their clinical translation to benefit patients.
在医学领域,临床医生能够将功能恢复和赋予特定细胞新功能用于治疗和疾病预防是很理想的。通过疾病、损伤或衰老而受损的细胞可以通过编程来恢复正常或丧失的功能,例如遗传性失明的视网膜细胞和阿尔茨海默病的神经元细胞。细胞也可以通过遗传编程获得新的功能,例如表达合成嵌合抗原受体的免疫细胞用于免疫治疗。此外,降低风险因子蛋白的水平或对其进行修饰可以减轻疾病的发展。目前,核酸作为一种多功能且有效的治疗手段,可用于实现这种细胞编程。在这篇综述中,我们从生物材料设计和递送来强调基于纳米生物材料的核酸治疗在细胞编程方面的最新进展,以及如何克服其临床转化的障碍,使患者受益。