Genome-wide profiling of circulating microRNAs in adolescent idiopathic scoliosis and their relation to spinal deformity severity, and disease pathophysiology

Abstract Adolescent Idiopathic Scoliosis (AIS) is the most common orthopedic condition requiring surgery, affecting 4% of adolescents. There is currently no proven method or prognostic test to identify symptomatic patients at risk of developing severe scoliosis who could benefit from growth-guided d...

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Main Authors: Nasrin Khatami, Iurie Caraus, Mahamuda Rahaman, Evguenia Nepotchatykh, Mohamed Elbakry, Wesam Elremaly, Anita Franco, Marie Beauséjour, Anne-Marie Laberge, Stefan Parent, Hubert Labelle, Carl-Éric Aubin, Jean Lachaine, Alain Moreau
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Language:English
Published: Nature Portfolio 2025-02-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-88985-3
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author Nasrin Khatami
Iurie Caraus
Mahamuda Rahaman
Evguenia Nepotchatykh
Mohamed Elbakry
Wesam Elremaly
Anita Franco
Marie Beauséjour
Anne-Marie Laberge
Stefan Parent
Hubert Labelle
Carl-Éric Aubin
Jean Lachaine
Alain Moreau
author_facet Nasrin Khatami
Iurie Caraus
Mahamuda Rahaman
Evguenia Nepotchatykh
Mohamed Elbakry
Wesam Elremaly
Anita Franco
Marie Beauséjour
Anne-Marie Laberge
Stefan Parent
Hubert Labelle
Carl-Éric Aubin
Jean Lachaine
Alain Moreau
author_sort Nasrin Khatami
collection DOAJ
description Abstract Adolescent Idiopathic Scoliosis (AIS) is the most common orthopedic condition requiring surgery, affecting 4% of adolescents. There is currently no proven method or prognostic test to identify symptomatic patients at risk of developing severe scoliosis who could benefit from growth-guided devices or minimally invasive non-fusion instrumentation surgeries. These innovative treatments must be performed at an early disease stage in younger patients to benefit from their growth potential. In this prospective cross-sectional study, we investigated the clinical utility of circulating microRNAs (miRNAs), an important class of small non-coding RNA, as biomarkers to predict the risk of developing severe scoliosis in AIS. Blood samples and clinical data were collected from 116 AIS patients who were followed until skeletal maturity and stratified according to their clinical outcome. Genome-wide expression profiling of miRNAs was performed with plasma obtained at the time of diagnosis of AIS (mean age of 13.3 ± 1.7 years with a mean Cobb angle of 24.4° ± 12.4°). This approach led to the identification of 15 circulating miRNAs that are upregulated in AIS patients who developed a severe scoliosis (Cobb angle ≥ 45°) at skeletal maturity compared to moderate and mild scoliosis groups (Cobb angle between 25°-44° and < 25° respectively). After optimization and the application of Random Forest Models a panel of six miRNAs (miR-1-3p, miR-19a-3p, miR-19b-3p, miR-133b, miR-143-3p, and miR-148b-3p) out of 15 led us to develop an algorithm predicting the risk of developing a severe scoliosis with great accuracy (100%), sensitivity (100%) and specificity (100%). Having a scoliosis predictive bioassay and decision-making tools to predict curve progression in order to find the best treatment plan will undoubtedly transform the orthopedic care system in the field of pediatric scoliosis by integrating innovative precision medicine approaches. In addition, investigation of genes targeted by these miRNAs could fill our gaps in our understanding of AIS pathogenesis and reveal new actionable targets.
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spelling doaj-art-40aebe4360cc4b8692d1fb0e30745a432025-08-20T02:43:10ZengNature PortfolioScientific Reports2045-23222025-02-0115111110.1038/s41598-025-88985-3Genome-wide profiling of circulating microRNAs in adolescent idiopathic scoliosis and their relation to spinal deformity severity, and disease pathophysiologyNasrin Khatami0Iurie Caraus1Mahamuda Rahaman2Evguenia Nepotchatykh3Mohamed Elbakry4Wesam Elremaly5Anita Franco6Marie Beauséjour7Anne-Marie Laberge8Stefan Parent9Hubert Labelle10Carl-Éric Aubin11Jean Lachaine12Alain Moreau13Viscogliosi Laboratory in Molecular Genetics of Musculoskeletal Diseases, Azrieli Research Center, CHU Sainte-JustineViscogliosi Laboratory in Molecular Genetics of Musculoskeletal Diseases, Azrieli Research Center, CHU Sainte-JustineViscogliosi Laboratory in Molecular Genetics of Musculoskeletal Diseases, Azrieli Research Center, CHU Sainte-JustineViscogliosi Laboratory in Molecular Genetics of Musculoskeletal Diseases, Azrieli Research Center, CHU Sainte-JustineViscogliosi Laboratory in Molecular Genetics of Musculoskeletal Diseases, Azrieli Research Center, CHU Sainte-JustineViscogliosi Laboratory in Molecular Genetics of Musculoskeletal Diseases, Azrieli Research Center, CHU Sainte-JustineViscogliosi Laboratory in Molecular Genetics of Musculoskeletal Diseases, Azrieli Research Center, CHU Sainte-JustineAzrieli Research Center, CHU Sainte-JustineMedical Genetics, Department of Pediatrics, CHU Sainte-Justine and Université de MontréalAzrieli Research Center, CHU Sainte-JustineAzrieli Research Center, CHU Sainte-JustineAzrieli Research Center, CHU Sainte-JustineFaculty of Pharmacy, Université de MontréalViscogliosi Laboratory in Molecular Genetics of Musculoskeletal Diseases, Azrieli Research Center, CHU Sainte-JustineAbstract Adolescent Idiopathic Scoliosis (AIS) is the most common orthopedic condition requiring surgery, affecting 4% of adolescents. There is currently no proven method or prognostic test to identify symptomatic patients at risk of developing severe scoliosis who could benefit from growth-guided devices or minimally invasive non-fusion instrumentation surgeries. These innovative treatments must be performed at an early disease stage in younger patients to benefit from their growth potential. In this prospective cross-sectional study, we investigated the clinical utility of circulating microRNAs (miRNAs), an important class of small non-coding RNA, as biomarkers to predict the risk of developing severe scoliosis in AIS. Blood samples and clinical data were collected from 116 AIS patients who were followed until skeletal maturity and stratified according to their clinical outcome. Genome-wide expression profiling of miRNAs was performed with plasma obtained at the time of diagnosis of AIS (mean age of 13.3 ± 1.7 years with a mean Cobb angle of 24.4° ± 12.4°). This approach led to the identification of 15 circulating miRNAs that are upregulated in AIS patients who developed a severe scoliosis (Cobb angle ≥ 45°) at skeletal maturity compared to moderate and mild scoliosis groups (Cobb angle between 25°-44° and < 25° respectively). After optimization and the application of Random Forest Models a panel of six miRNAs (miR-1-3p, miR-19a-3p, miR-19b-3p, miR-133b, miR-143-3p, and miR-148b-3p) out of 15 led us to develop an algorithm predicting the risk of developing a severe scoliosis with great accuracy (100%), sensitivity (100%) and specificity (100%). Having a scoliosis predictive bioassay and decision-making tools to predict curve progression in order to find the best treatment plan will undoubtedly transform the orthopedic care system in the field of pediatric scoliosis by integrating innovative precision medicine approaches. In addition, investigation of genes targeted by these miRNAs could fill our gaps in our understanding of AIS pathogenesis and reveal new actionable targets.https://doi.org/10.1038/s41598-025-88985-3Adolescent idiopathic scoliosisCirculating microRNAsSpinal deformity severityDisease pathophysiologyDiagnostic and prognostic biomarkersMachine learning
spellingShingle Nasrin Khatami
Iurie Caraus
Mahamuda Rahaman
Evguenia Nepotchatykh
Mohamed Elbakry
Wesam Elremaly
Anita Franco
Marie Beauséjour
Anne-Marie Laberge
Stefan Parent
Hubert Labelle
Carl-Éric Aubin
Jean Lachaine
Alain Moreau
Genome-wide profiling of circulating microRNAs in adolescent idiopathic scoliosis and their relation to spinal deformity severity, and disease pathophysiology
Scientific Reports
Adolescent idiopathic scoliosis
Circulating microRNAs
Spinal deformity severity
Disease pathophysiology
Diagnostic and prognostic biomarkers
Machine learning
title Genome-wide profiling of circulating microRNAs in adolescent idiopathic scoliosis and their relation to spinal deformity severity, and disease pathophysiology
title_full Genome-wide profiling of circulating microRNAs in adolescent idiopathic scoliosis and their relation to spinal deformity severity, and disease pathophysiology
title_fullStr Genome-wide profiling of circulating microRNAs in adolescent idiopathic scoliosis and their relation to spinal deformity severity, and disease pathophysiology
title_full_unstemmed Genome-wide profiling of circulating microRNAs in adolescent idiopathic scoliosis and their relation to spinal deformity severity, and disease pathophysiology
title_short Genome-wide profiling of circulating microRNAs in adolescent idiopathic scoliosis and their relation to spinal deformity severity, and disease pathophysiology
title_sort genome wide profiling of circulating micrornas in adolescent idiopathic scoliosis and their relation to spinal deformity severity and disease pathophysiology
topic Adolescent idiopathic scoliosis
Circulating microRNAs
Spinal deformity severity
Disease pathophysiology
Diagnostic and prognostic biomarkers
Machine learning
url https://doi.org/10.1038/s41598-025-88985-3
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