Extracellular Vesicles as Biomarkers and Therapeutic Tools: From Pre-Clinical to Clinical Applications

SIMPLE SUMMARY: Extracellular vesicles (EVs) are membrane-bound vesicles released by all cell types, differing in biogenesis, physical characteristics, and contents. Due to their central role in intercellular communication and their variable cargo, EVs are involved in several biological processes. The possibility of isolating them from different biofluids makes EVs valuable biomarkers to be analyzed for the diagnosis or prognosis of several conditions. Moreover, these natural nanoparticles have been investigated as therapeutic tools in many pathological conditions. In this context, EVs have shown innate immunosuppressive and anti-inflammatory properties when isolated from stem/progenitor cells and have also been considered vehicles to be edited for drug delivery. The aim of the review is to report some of the pre-clinical and clinical studies distinguishing those in which EVs have been examined as biomarkers from those in which they have been used as therapeutics. ABSTRACT: Extracellular vesicles (EVs) are ubiquitous masters of intercellular communication, being detectable in tissues, circulation, and body fluids. Their complex cargo reflects the (patho)physiologic status of the cells from which they originate. Due to these properties, the potential of EVs, and in particular exosomes, to serve as biomarkers or therapeutics has grown exponentially over the past decade. On one side, numerous studies have demonstrated that EV-associated nucleic acids and proteins are implicated in cancer progression, as well as neurodegenerative, infectious, and autoimmune disorders. On the other, the therapeutic use of EVs secreted by various cell types, and in particular stem/progenitor cells, present significant advantages in comparison to the corresponding parental cells, such as the less complex production and storage conditions. In this review, we examine some of the major pre-clinical studies dealing with EVs and exosomes, that led to the development of numerous completed clinical trials.