Lee Eun Chae, Choi Dongsic, Lee Dong-Hun, Oh Jae Sang
Department of Medical Sciences, Graduate School, The Catholic University of Korea, Seoul 06591, Republic of Korea.
Department of Biochemistry, Soonchunhyang University College of Medicine, Cheonan 31151, Republic of Korea.
Int J Mol Sci. 2025 Mar 28;26(7):3137. doi: 10.3390/ijms26073137.
The development of targeted drugs for diseases of the central nervous system (CNS) is a significant challenge due to the structural complexity and functional specificities of these systems. Recently, exosomes have emerged as a promising therapeutic platform, given their unique capacity to traverse the blood-brain barrier and deliver bioactive molecules to target cells. This review examines recent advances in exosome research with a particular focus on CNS diseases, emphasizing their role as carriers of therapeutic cargo, including proteins, RNAs, and lipids. Nevertheless, significant challenges remain before exosome-based therapies can be translated from preclinical research to clinical applications. These include the need for scalable production and standardized isolation methods. Despite these hurdles, ongoing studies continue to shed light on the mechanisms of exosome-mediated neuroprotection and neurodegeneration. This paves the way for innovative therapeutic strategies to address CNS disorders.
由于中枢神经系统（CNS）的结构复杂性和功能特异性，开发针对中枢神经系统疾病的靶向药物是一项重大挑战。最近，外泌体已成为一个有前景的治疗平台，因为它们具有穿越血脑屏障并将生物活性分子递送至靶细胞的独特能力。本综述考察了外泌体研究的最新进展，特别关注中枢神经系统疾病，强调它们作为治疗性货物（包括蛋白质、RNA和脂质）载体的作用。然而，在基于外泌体的疗法能够从临床前研究转化为临床应用之前，仍然存在重大挑战。这些挑战包括需要可扩展的生产和标准化的分离方法。尽管存在这些障碍，但正在进行的研究继续揭示外泌体介导的神经保护和神经退行性变的机制。这为解决中枢神经系统疾病的创新治疗策略铺平了道路。
