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World J Stem Cells. Mar 26, 2026; 18(3): 116226
Published online Mar 26, 2026. doi: 10.4252/wjsc.v18.i3.116226
Stem cell derived exosomes: Emerging cell-free therapeutics for neurodegenerative disorders
Mahmood S Choudhery, Taqdees Arif, Ruhma Mahmood, David T Harris
Mahmood S Choudhery, Taqdees Arif, Department of Human Genetics and Molecular Biology, University of Health Sciences, Lahore 54000, Punjab, Pakistan
Ruhma Mahmood, Department of Pathology, Allama Iqbal Medical College, Lahore 54000, Punjab, Pakistan
David T Harris, Department of Immunobiology, University of Arizona Health Sciences Biorepository, The University of Arizona, Tucson, AZ 85721, United States
Author contributions: Choudhery MS, Arif T, and Mahmood R designed the contents of the manuscript, prepared, designed and modified the figures; Choudhery MS and Arif T wrote the original version of manuscript; Arif T and Mahmood R revised the manuscript; Choudhery MS and Harris DT critically reviewed the manuscript. All authors reviewed the manuscript.
Conflict-of-interest statement: All the authors report no relevant conflicts of interest for this article.
Corresponding author: Mahmood S Choudhery, PhD, Associate Professor, Department of Human Genetics and Molecular Biology, University of Health Sciences, Khayaban-e-Jamia Punjab, Lahore 54000, Punjab, Pakistan. ms20031@yahoo.com
Received: November 6, 2025
Revised: December 12, 2025
Accepted: February 5, 2026
Published online: March 26, 2026
Processing time: 140 Days and 1.5 Hours
Abstract

Neurodegenerative disorders such as Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis represent a significant global health challenge with limited therapeutic options. In recent years, stem cell-based therapies have shown promise in promoting neural repair and modulating disease progression. However, concerns related to immune rejection, tumorigenicity, and ethical considerations have limited their clinical application. As an alternative, exosomes derived from stem cells have emerged as a novel, acellular therapeutic strategy. These nanoscale extracellular vesicles carry a rich cargo of proteins, lipids, and nucleic acids, capable of modulating neuroinflammation, promoting neuroprotection, and enhancing tissue repair. Their ability to cross the blood-brain barrier and their low immunogenicity make them especially attractive for treating central nervous system disorders. This review highlights the therapeutic potential of stem cell-derived exosomes in the management of neurodegenerative diseases, discussing their mechanisms of action, current research progress, and future clinical applications. The development of exosome-based therapies marks a significant step toward safe, effective, and cell-free neurodegeneration.

Keywords: Neurodegenerative diseases; Alzheimer’s disease; Parkinson’s disease; Stroke; Exosomes; Acellular therapy

Core Tip: Neurodegenerative disorders including Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, and amyotrophic lateral sclerosis, are progressive and irreversible conditions marked by neuronal loss, misfolded protein accumulation, and severe cognitive or motor decline. Current therapies cannot halt neuronal death, and stem cell transplantation is limited by the blood-brain barrier. Exosomes, nanoscale vesicles secreted by cells, overcome this limitation by naturally crossing the blood-brain barrier, carrying proteins, nucleic acids, and lipids with neuroprotective potential. Their role in diagnosis, biomarker discovery, and targeted drug delivery highlights exosomes as promising acellular therapeutics for neurodegenerative disorders, with growing clinical interest and translational potential.