Clinical Applications of Extracellular Vesicles: Promises and Pitfalls

Extracellular vesicles (EVs) are membrane-bound nanoparticles released by almost all cell types into the extracellular space, acting as important mediators of intercellular communication by transferring proteins, lipids, and nucleic acids horizontally. EVs are generally classified into small EVs (<200 nm), medium/large EVs (>200 nm), microvesicles, and apoptotic bodies, with current classification methods focusing on physical properties, molecular composition, and cellular origin, as detailed in the MISEV2023 guidelines. EVs are highly promising for diagnostic and therapeutic applications due to their intrinsic biocompatibility, stability in biological fluids, capacity to carry diverse molecular cargo, and potential for drug delivery and functionalization to enable targeted delivery and tissue repair. This narrative review discusses the emerging roles of EVs across various medical fields, including obstetrics and gynecology, ophthalmology, otorhinolaryngology, urology, oncology, orthopedics, neurology, immunology, wound healing, chronic pain management, dermatology, and cardiology. In each discipline, EVs show potential as biomarkers for diagnosing physiological or pathological conditions and as carriers for targeted drug delivery and regenerative treatments. Exosomes, a major type of small EVs, have especially attracted attention as versatile nanocarriers for precision medicine. However, translation into clinical practice requires addressing key pitfalls, including the standardization of isolation and characterization protocols, dose definition, GMP-compliant large-scale production, and regulatory approval. Ongoing interdisciplinary collaboration across disciplines and thorough clinical testing will be essential to unlock the full biomedical potential of EVs and establish them as transformative tools in personalized healthcare.

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Publication Details

Journal
International Journal of Molecular Sciences
Published
2026-02-03
DOI
https://doi.org/10.3390/ijms27031509
Primary Topic
Extracellular vesicles in disease
Type
article
Field-Weighted Citation Impact
0.00

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article

Clinical Applications of Extracellular Vesicles: Promises and Pitfalls

Pierre Rocheteau, Ariana DeMers, Mary A. Ambach, Petar Brlek et al.
International Journal of Molecular Sciences
Extracellular vesicles in disease
article

Clinical Applications of Extracellular Vesicles: Promises and Pitfalls

Pierre Rocheteau, Ariana DeMers, Mary A. Ambach, Petar Brlek, Ana Medić Flajšman, Dragan Primorac, Ilona Uzielienè, Don Buford, Adelina Hrkać, Carlo Tremolada, Martina Glavan, Eiva Bernotienė, Dimitrios Tsoukas, Wei Seong Toh, Raminta Vaičiulevičiūtė, Neep Patel, Lindsay Williams, Massimo Allegri, EV Badiavas, Dimitrios Kouroupis, Gordana Kalan Živčec, Jolita Pachaleva, Cole Conforti, A. Mobasheri, Luka Bulić, Miomir Knežević, Kristiana Barbato, Nenad Hrvatin, Tamara Knežević, Vasiliki E. Kalodimou, Isabella Demirdjian Guanche, Žan Fortuna, Petar Projić, Lara Oprešnik, R. Peter Schnorr, Jana Mešić, D. Wood Kimbrough, Danijela Klarić, Vedrana Škaro, Nancy Duarte Delgado, Ijeoma Oleru, Romina Milanič, Blaž Curk, Johannes Brachmann
article en

Abstract

Extracellular vesicles (EVs) are membrane-bound nanoparticles released by almost all cell types into the extracellular space, acting as important mediators of intercellular communication by transferring proteins, lipids, and nucleic acids horizontally. EVs are generally classified into small EVs (<200 nm), medium/large EVs (>200 nm), microvesicles, and apoptotic bodies, with current classification methods focusing on physical properties, molecular composition, and cellular origin, as detailed in the MISEV2023 guidelines. EVs are highly promising for diagnostic and therapeutic applications due to their intrinsic biocompatibility, stability in biological fluids, capacity to carry diverse molecular cargo, and potential for drug delivery and functionalization to enable targeted delivery and tissue repair. This narrative review discusses the emerging roles of EVs across various medical fields, including obstetrics and gynecology, ophthalmology, otorhinolaryngology, urology, oncology, orthopedics, neurology, immunology, wound healing, chronic pain management, dermatology, and cardiology. In each discipline, EVs show potential as biomarkers for diagnosing physiological or pathological conditions and as carriers for targeted drug delivery and regenerative treatments. Exosomes, a major type of small EVs, have especially attracted attention as versatile nanocarriers for precision medicine. However, translation into clinical practice requires addressing key pitfalls, including the standardization of isolation and characterization protocols, dose definition, GMP-compliant large-scale production, and regulatory approval. Ongoing interdisciplinary collaboration across disciplines and thorough clinical testing will be essential to unlock the full biomedical potential of EVs and establish them as transformative tools in personalized healthcare.

International Journal of Molecular SciencesVol. 27(3)
Lee College (US), City Of Hope National Medical Center (US), Pennsylvania State University (US), National Forensic Sciences University (IN), University of Miami (US), University of Rijeka (HR), University of Liège (BE), Sun Yat-sen University (CN), National University of Singapore (SG), University of Pittsburgh (US), University of Zagreb (HR), European University Cyprus (CY), Yale University (US), University of New Haven (US), Sana Kliniken Duisburg (DE), University of Miami Health System (US), State Research Institute Centre for Innovative Medicine (LT), City of Hope (US), Adc Therapeutics (Switzerland) (CH), Hôpital de Morges (CH), The First Affiliated Hospital, Sun Yat-sen University (CN), Mitera Hospital (GR), Regenerative Medicine Institute (MX), Methodist Sports Medicine (US), Educell (SI), Miami Transplant Institute (US), Beckman Research Institute, University of Osijek (HR), University of Split (HR), University of Oulu (FI)
University of Miami, Lietuvos Mokslo Taryba, National Institutes of Health, Directorate for Biological Sciences, Leonard M. Miller School of Medicine, Leonard M. Miller School of Medicine, University of Miami, HORIZON EUROPE Framework Programme
Openalex Percentile: Top 69%
Extracellular vesicles in disease
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