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Nanotechnology-Driven Drug-Delivery Systems: Mechanistic Insights for Pediatric Autism Treatment in 2026
Mahboubeh Atashgahi1, Fatemeh Madani2, Thomas J Webster3,4,5
1Department of Surgery, Royal Children's Hospital, Melbourne, VIC, Australia.
Insights
Nanomedicine offers new ways to treat autism spectrum disorder (ASD) by overcoming the blood-brain barrier (BBB) for targeted drug delivery and developing advanced diagnostic tools.
Area of Science:
- Neuroscience
- Developmental Pediatrics
- Nanomedicine
Background:
- Autism spectrum disorder (ASD) presents significant challenges in pediatric care, with current treatments focusing on symptom management rather than core mechanisms.
- Abnormalities in synaptic function, neuroinflammation, and the gut-brain axis are implicated in ASD pathogenesis, indicating multiple therapeutic targets.
- The blood-brain barrier (BBB) poses a major obstacle for central nervous system (CNS) drug delivery in ASD, yet its properties also present opportunities.
Purpose of the Study:
- To review the clinical and mechanistic aspects of ASD relevant to therapeutic interventions.
- To explore the role of nanomedicine and nanoparticles in addressing ASD challenges.
- To provide a framework for nano-enabled interventions in ASD.
Main Methods:
- Literature review of clinical manifestations, mechanistic pathways, and BBB biology in ASD.
- Analysis of nanoparticle-based methodologies for neurological applications.
- Integration of developmental perspectives for nano-interventions.
Main Results:
- Nanoparticles show promise for targeted drug delivery to the brain in neurological disorders.
- Nanomedicine can aid in developing advanced ASD models and diagnostic/theranostic tools.
- The BBB's unique characteristics can be leveraged for effective CNS drug delivery.
Conclusions:
- Nanomedicine presents a promising frontier for developing novel therapeutic strategies for ASD.
- Targeted nanoparticle delivery systems can potentially overcome BBB limitations for treating ASD.
- Further research integrating nanomedicine with developmental insights is crucial for advancing ASD care.
Abstract:
Autism spectrum disorder (ASD) is a diverse neurodevelopmental disorder that commences in early childhood, characterized by enduring social-communication challenges, limited interests, and behavioral rigidity. The management of ASD continues to pose significant difficulties in pediatric practice. Converging evidence implicates abnormalities in synaptic scaffolding and transmission, excitation-inhibition imbalance, mTOR/PI3K-AKT signaling, neuroinflammation, gut-brain axis, and metabolic disturbances, highlighting multiple cellular and molecular targets for potential therapeutic development. Nonetheless, contemporary care is primarily characterized by non-pharmacological interventions and symptomatic pharmacological treatments, with a scarcity of strategies that alter fundamental mechanisms. The blood-brain barrier (BBB) is a significant hurdle to overcome - its structure, transport routes, and context-specific dysfunction in ASD both limit and create opportunities for central nervous system (CNS) drug delivery at the same time. In this context, nanomedicine presents novel opportunities and nanoparticles are currently emerging for various neurological applications in preclinical and clinical studies. They can be designed for targeted drug delivery to the brain, for the development of sophisticated ASD models and for diagnostic and theranostic purposes. This review incorporates the clinical manifestations, mechanistic pathways, BBB biology, and nanoparticle-based methodologies to provide a developmentally informed framework for nano-enabled interventions in ASD.
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