Extracellular Vesicles in Amyotrophic Lateral Sclerosis

Gavin McCluskey, Karen E. Morrison, Colette Donaghy, Frederique Rene, William Duddy, Stephanie Duguez, Pilar Maria Ferraro (Editor)

Research output: Contribution to journalReview articlepeer-review

8 Citations (Scopus)
32 Downloads (Pure)


Amyotrophic Lateral Sclerosis is a progressive neurodegenerative disease and is the most common adult motor neuron disease. The disease pathogenesis is complex with the perturbation of multiple pathways proposed, including mitochondrial dysfunction, RNA processing, glutamate excitotoxicity, endoplasmic reticulum stress, protein homeostasis and endosomal transport/extracellular vesicle (EV) secretion. EVs are nanoscopic membrane-bound particles that are released from cells, involved in the intercellular communication of proteins, lipids and genetic material, and there is increasing evidence of their role in ALS. After discussing the biogenesis of EVs, we review their roles in the propagation of pathological proteins in ALS, such as TDP-43, SOD1 and FUS, and their contribution to disease pathology. We also discuss the ALS related genes which are involved in EV formation and vesicular trafficking, before considering the EV protein and RNA dysregulation found in ALS and how these have been investigated as potential biomarkers. Finally, we highlight the potential use of EVs as therapeutic agents in ALS, in particular EVs derived from mesenchymal stem cells and EVs as drug delivery vectors for potential treatment strategies.
Original languageEnglish
Article number121
Pages (from-to)1-20
Number of pages20
Issue number1
Early online date31 Dec 2022
Publication statusPublished online - 31 Dec 2022


  • Review
  • exosomes
  • extracellular vesicles
  • amyotrophic lateral sclerosis
  • motor neuron disease


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