Therapeutic Potential of Extracellular Vesicles: From Biogenesis, Isolation and Molecular Characterization to Addressing Translational Gaps and Regulatory Barriers
Extracellular vesicles (EVs) have emerged as essential mediators of intercellular communication, transporting a complex repertoire of lipids, proteins, and nucleic acids that mirror the physiological and pathological status of their parent cells. This review provides a comprehensive overview of EVs from their biogenesis and molecular composition to their translational potential in human disease. This review outlines the major classes of EVs, including exosomes, microvesicles, apoptotic bodies, and oncosomes, together with recent developments in their isolation, molecular characterization, and omics-based profiling. Special focus is given to the role of EVs in viral infection, inflammation, and immune regulation, as well as their contribution to disease development and cancer biology. Moreover, we highlight the emerging clinical applications of mesenchymal stem cell-derived EVs (MSC-EVs) in regenerative medicine and oncology, alongside the therapeutic modulation of EV signaling by photobiomodulation (PBM). Finally, we address key translational challenges related to standardization, scalability, and regulatory validation. As exosome-based therapeutics fall under strict FDA and EMA oversight, their translation further depends on harmonized quality controls and robust safety evaluation. By integrating molecular mechanisms with clinical applications, this review emphasizes the transformative potential of EVs as next-generation diagnostic and therapeutic tools in precision medicine.
Authors
- Pierre Rocheteau
- Ariana DeMers
- Mary A. Ambach (ORCID: https://orcid.org/0000-0003-2978-8594)
- Petar Brlek (ORCID: https://orcid.org/0000-0001-8022-4095)
- Ana Medić Flajšman
- Dragan Primorac (ORCID: https://orcid.org/0000-0001-5565-080X)
- Ilona Uzielienè (ORCID: https://orcid.org/0000-0001-5484-9163)
- Don Buford (ORCID: https://orcid.org/0000-0002-9547-4422)
- Adelina Hrkać
- Carlo Tremolada (ORCID: https://orcid.org/0000-0003-1331-5995)
- Martina Glavan (ORCID: https://orcid.org/0000-0001-6710-905X)
- Eiva Bernotienė (ORCID: https://orcid.org/0000-0002-1529-5513)
- Dimitrios Tsoukas (ORCID: https://orcid.org/0000-0001-7966-6670)
- Wei Seong Toh (ORCID: https://orcid.org/0000-0001-9147-6423)
- Raminta Vaičiulevičiūtė (ORCID: https://orcid.org/0000-0002-4315-7783)
- Neep Patel (ORCID: https://orcid.org/0000-0001-8241-0959)
- Lindsay Williams (ORCID: https://orcid.org/0000-0002-9877-2935)
- Massimo Allegri (ORCID: https://orcid.org/0000-0002-8201-980X)
- EV Badiavas
- Dimitrios Kouroupis (ORCID: https://orcid.org/0000-0002-3892-9013)
- Gordana Kalan Živčec
- Jolita Pachaleva (ORCID: https://orcid.org/0000-0001-8118-6377)
- Cole Conforti
- Luka Bulić (ORCID: https://orcid.org/0000-0001-6522-891X)
- Miomir Knežević
- Kristiana Barbato
- Nenad Hrvatin (ORCID: https://orcid.org/0009-0005-8797-3239)
- Tamara Knežević
- Vasiliki E. Kalodimou
- Isabella Demirdjian Guanche
- Žan Fortuna
- Petar Projić
- Lara Oprešnik
- Jana Mešić
- Danijela Klarić
- Vedrana Škaro
- Nancy Duarte Delgado
- Ijeoma Oleru
- Romina Milanič
- Blaž Curk
- Johannes Brachmann
- R. Peter Schnorr
- D. Wood Kimbrough
- Ali Mobasheri
Institutions
- 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)
Publication Details
- Journal
- International Journal of Molecular Sciences
- Published
- 2026-02-09
- DOI
- https://doi.org/10.3390/ijms27041676
- Primary Topic
- Extracellular vesicles in disease
- Type
- article
- Field-Weighted Citation Impact
- 0.00