Bridging development and disease: the potential of LncRNAs as biomarkers and therapeutics in pediatric neurological disorders.

PMID 42071079 | DOI 10.1038/s41390-026-04954-0 · Pediatric research · 2026 · View on PubMed ↗

Long non-coding RNAs (lncRNAs) represent a large family of RNA transcripts that are longer than 200 nucleotides and don't code for proteins. They have recently emerged as key regulators of gene expression which modulate a wide range of biological processes from the early periods of embryonic development to disease progression. Therefore, they represent a cornerstone in pediatric neurology, possibly through orchestrating neural differentiation, synaptic plasticity, and neural circuit maturation. Furthermore, they significantly contribute to the pathogenesis of the neurodevelopmental disorders such as autism spectrum conditions, epilepsy, and delays in the neurodevelopmental milestones. The focus of this review article is on the utility of lncRNAs as biomarkers and therapies for neurological pediatric disorders, the basis for this and therapeutic strategies. In addition, this review article may represent a trial to explore how lncRNAs can establish both direct and indirect connections between the genetic, epigenetic, and environmental factors. Consequently, this may open avenues for innovative diagnostic biomarkers and the development of novel therapeutic strategies for pediatric neurological disorders. IMPACT: This article elucidates the multifaceted roles of lncRNAs in bridging the normal neural development with the disease states. This article highlights the current limitations/barriers to the clinical translation of lncRNA-based therapies in pediatrics and provides emerging solutions. Guides how lncRNAs establish connections between the genetic, epigenetic, and environmental factors. This may open avenues for innovative diagnostic biomarkers and novel therapeutic strategies for pediatric neurological disorders.