Journal September 26, 2026 5 min read

What does the research say about exosomes?

Exosomes are small extracellular vesicles that carry proteins, lipids, and nucleic acids between cells. Research into their biology spans in vitro, animal, and early human studies, with applications explored in cancer, w

peptides-pro Editorial

What are exosomes?

Exosomes are a subclass of extracellular vesicles, typically 30–150 nm in diameter, released by most cell types. They are formed through the endosomal pathway and carry a cargo of proteins, lipids, and nucleic acids that can be transferred to recipient cells. In the research literature, they are sometimes referred to as small extracellular vesicles (sEVs) or simply as exosomal fractions. Unlike whole cells, exosomes are acellular and cannot replicate. Their molecular composition varies by cell of origin and physiological state, and no single sequence, formula, or molecular weight defines the class as a whole.

Much of the work on exosomes centres on their role in intercellular communication, particularly via receptor-mediated signalling pathways such as EGFR, and on their use as delivery vehicles or biomarkers in disease models.

Research Studies

Exosomal hsa-miR-221-5p and keratinocyte LL-37 in melanoma progression

In clinical specimens, spatial suppression of the antimicrobial peptide LL-37 was observed in tumor-adjacent epidermis compared with distal regions. Melanoma-derived exosomes suppressed keratinocyte LL-37 expression, an effect driven in part by exosomal hsa-miR-221-5p through impaired sustained EGFR signalling. Functionally, keratinocyte-derived LL-37 curtailed melanoma cell migration, invasion, and epithelial–mesenchymal transition, restrained tumour growth in vivo, and was associated with a tumour immune microenvironment enriched in M1 macrophages [2].

FGF21-modified ADSC exosomes and fibroblast glycolysis in wound healing

Adipose-derived mesenchymal stem cells (ADSCs) overexpressing fibroblast growth factor 21 (FGF21) were established using genetic engineering, and FGF21-enriched exosomes (Exo@FGF21) were isolated and characterised by transmission electron microscopy and nanoparticle tracking analysis. The study aimed to elucidate mechanisms by which these engineered exosomes promote wound healing, with a focus on activating fibroblast glycolysis through AMPK/mTOR signalling [3].

Cerebrolysin-treated endothelial cell exosomes and cerebral endothelial permeability

Using an in vitro endothelial permeability assay, exosomes released by Cerebrolysin-treated cerebral endothelial cells (Cerebro-Exos) were tested for effects on dysfunctional cerebral endothelial cells. Stoichiometric analysis showed marked differences in cargo profiles between Cerebro-Exos and exosomes from untreated cells (Naïve-Exos); Cerebro-Exos were highly enriched with metabolic and tight junction-related proteins. Cerebro-Exos had a superior effect compared to Naïve-Exos on restoring endothelial cell integrity impaired by fibrin and tissue plasminogen activator (tPA) [4].

Urinary exosome podocyte markers in diabetic kidney disease

A clinical study is examining podocyte-specific injury markers—nephrin, podocalyxin, and Wilms' tumour (WT-1) protein—in urinary exosomes from African American Veterans with albuminuric stage 2 and 3 chronic diabetic kidney disease. Participants are treated with empagliflozin, finerenone, or combination therapy. Secondary objectives include correlating changes in exosome-based podocyte-specific proteins with standardised biomarkers of kidney injury (urine albumin/creatinine ratio and estimated GFR) and with systemic inflammatory markers such as interleukins (IL-1, IL-6, IL-12), hs-CRP, and arterial stiffness measurements [1].

Neuronal exosomes and exercise-induced anxiolysis

A study is investigating the brain-based biological mechanistic pathway by which exercise exerts anxiolytic effects. The investigators measure brain-derived markers of mitochondrial metabolism (acetyl-L-carnitine) and inflammation (interleukin-6) using technology to isolate neuronal exosomes [7].

Umbilical cord blood-derived stem cells and exosomes in autism spectrum disorder

A 12-month study is testing whether AdiaVita (umbilical cord blood-derived stem cells and exosomes) plus glutathione improves autism symptoms more than glutathione alone in children ages 3–12. Approximately 100 children with confirmed autism are randomly assigned to receive monthly intravenous AdiaVita plus intravenous glutathione for 3 months, or intravenous glutathione plus a saline placebo infusion for 2 months. Both groups also use a topical glutathione spray twice daily at home. Symptoms are tracked with the Autism Treatment Evaluation Checklist (ATEC), completed by a parent and by a therapist or teacher. Safety is monitored at clinic visits through Month 12 [5].

Wharton's jelly exosomes with autologous bone marrow cells in liver cirrhosis

A clinical study includes patients with liver cirrhosis irrespective of stage, excluding those with active tumour or other major health issues. The protocol uses autologous bone marrow-derived mononuclear cells harvested from the same patient under local anaesthesia, followed by cell concentration and viability testing. The final product is combined with a small volume (2 cc) of Wharton's jelly exosomes (20 billion) and administered intravenously [8].

Regenerative medicine approaches to craniofacial and corneal neuropathic pain

A review discusses regenerative medicine approaches to craniofacial and corneal neuropathic pain, conditions characterised by persistent pain refractory to conventional therapies. These disorders involve peripheral nerve injury, neuroinflammation, and maladaptive central sensitisation within trigeminal pathways. The review emphasises the need for mechanism-based, patient-stratified treatment strategies and describes a paradigm shift from symptom suppression toward structural nerve repair and functional restoration [6].

Exosome-based biomarkers and therapeutic delivery in other models

Additional studies have explored exosomes in various contexts. In pancreatic cancer, cancer-associated fibroblast-derived exosomes delivering Snail were investigated for their role in promoting cancer stemness and gemcitabine resistance via the SOCS6/HIF-1α axis [9]. In wound healing, a chitosan hydrogel loaded with mesenchymal stem cell-derived exosomes accelerated wound closure and enhanced tissue regeneration in a full-thickness porcine skin defect model [10]. A combination of LL-37 and adipose-derived stem cells was studied for accelerating diabetic wound repair through NRROS-mediated crosstalk between TGF-β/SMAD and Hippo/TEAD1 axes [11]. In glioma, surface-engineered GE11-functionalised exosomes were developed for EGFR-targeted delivery of peonidin and suppression of SNAI1-mediated epithelial–mesenchymal transition [12].

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