Harnessing MSC‑derived exosomes to modulate the pathophysiology of ASD: Recent advances and therapeutic implications (Review)
Year: 2026
Type: article
Abstract: Autism spectrum disorder (ASd) is a complex neurodevelopmental condition characterized by marked genetic heterogeneity and diverse environmental influences.current treatment approaches focus on symptom management, with only a limited number of effective interventions targeting the underlying causes.Recently, mesenchymal stem cells (MScs) and their derived exosomes (MSc-Exos) have emerged as promising candidates for ASd therapy owing to their potent immunomodulatory, neuroprotective and targeted delivery properties.The present review discusses the functions of MSC-Exos and their potential use in ASD.MSC-Exos improve neuroinflammation, enhance synaptic plasticity and restore neural network function by delivering bioactive molecules.Moreover, MSC-Exos exhibit a low immunogenicity, a favorable safety profile and scalability for clinical production.Despite promising results however, clinical trials continue to face challenges, particularly in standardizing the isolation, characterization, dosing and administration routes of exosomes.In addition, significant challenges persist in production processes, quality control and the elucidation of the mechanisms of action.In conclusion, MSC-Exos represent a groundbreaking, cell-free therapeutic strategy with substantial potential to target the core pathophysiology of ASD.In the future, multicenter randomized controlled trials and interdisciplinary collaborations will be crucial for translating preclinical findings into the development of effective and transformative therapies for ASD. Contents1.The concept of autism spectrum disorder and recent progress 2. Molecular genetics and neuropathology of ASD 3. Mesenchymal stem cells and their derived exosomes in ASd: Research and applications 4. Clinical progress of stem cells and exosomes in ASD 5. Conclusion 6. Future perspectives and application directions 1.The concept of autism spectrum disorder and recent progress Core symptoms and current epidemiological status.Autism spectrum disorder (ASd) encompasses a group of pervasive neurodevelopmental conditions characterized by a broad spectrum of symptoms, broadly categorized into core and associated symptoms.Core symptoms include persistent deficits in social interaction and communication, as well as restricted and repetitive patterns of behavior and interests that significantly impair daily functioning.By contrast, associated symptoms encompass attention deficits, mood disturbances, sensory processing abnormalities, sleep disorders, language impairment, anxiety, epilepsy, mania and self-injurious behaviors.Core symptoms typically emerge in early childhood and Harnessing MSC-derived exosomes to modulate the pathophysiology of ASD: Recent advances and therapeutic implications (Review)
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
OpenAlex ID: W7124456253