MiR-182-5p, miR-122-5p and hsa-piR-28004 as indicators of disease and treatment in pediatric familial hypercholesterolemia

This is the first comprehensive profiling of both microRNAs (miRNAs) and piwi-interacting RNAs (piRNAs) in children and adolescents with heterozygous familial hypercholesterolemia (heFH). Circulating miR-182-5p, miR-122-5p and hsa-piR-28004 are potential early indicators of disease and treatment response in pediatric heFH. MiRNAs identified to be associated with atherosclerosis in adults are not robustly expressed in pediatric heFH, underscoring age-specific molecular differences. These findings support the potential of non-coding RNAs (ncRNAs) as non-invasive biomarkers for improved diagnosis, monitoring, and personalized risk assessment in children and adolescents with heFH beyond traditional lipid metrics.