Engineering Exosomes for CNS Disorders: Advances, Challenges, and Therapeutic Potential
Eun Chae Lee, Dongsic Choi, Dong-Hun Lee, Jae Sang Oh
Abstract
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.