Whole-exome sequencing screening for candidate genes and potential pathogenic variants associated with early-onset high myopia in 47 Chinese families

Abstract Early-onset high myopia (eoHM) occurs before school age and is an ideal model for monogenic studies of high myopia due to minimal environmental influence. This study screened for genes and variants associated with eoHM in 47 unrelated Chinese patients with eoHM. Protein–protein interaction...

Full description

Saved in:
Bibliographic Details
Main Authors: Xue Rui, Huiping Li, Runqing Ma, Shangying Yang, Yuanyuan lian, Wanyu Cheng, Meijiao Ma, Weining Rong, Xunlun Sheng
Format: Article
Language:English
Published: Nature Portfolio 2025-04-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-025-95574-x
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850265396340326400
author Xue Rui
Huiping Li
Runqing Ma
Shangying Yang
Yuanyuan lian
Wanyu Cheng
Meijiao Ma
Weining Rong
Xunlun Sheng
author_facet Xue Rui
Huiping Li
Runqing Ma
Shangying Yang
Yuanyuan lian
Wanyu Cheng
Meijiao Ma
Weining Rong
Xunlun Sheng
author_sort Xue Rui
collection DOAJ
description Abstract Early-onset high myopia (eoHM) occurs before school age and is an ideal model for monogenic studies of high myopia due to minimal environmental influence. This study screened for genes and variants associated with eoHM in 47 unrelated Chinese patients with eoHM. Protein–protein interaction (PPI) network analysis was conducted to detect interactions among candidate genes, and protein–protein docking was performed. In 28 patients (28/47, 59.6%), 32 potential pathogenic variants in 22 candidate genes were identified, including 24 novel variants. Among these 28 patients, 64.3% (18/28) carried pathogenic variants in RetNet genes. Of these, 12 patients (42.9%, 12/28) had pathogenic variants in six known genes (TSPAN12, CACNA1F, USH2A, RPGR, COL2A1, and COL11A1), which are responsible for retinal dystrophy and Stickler syndrome associated with eoHM. Additionally, 7 patients (25.0%, 7/28) carried pathogenic variants in seven candidate genes for ocular disease (POLA1, TMEM231, HK1, GSN, COL5A1, CRYBB3, and WDR), which were identified as potentially pathogenic in Chinese eoHM patients for the first time. Phenotype analysis showed that 11 patients presented with only high myopia, 10 patients had inherited retinal diseases (IRDs) with eoHM, and 7 patients had ocular-only Stickler syndrome (Ocular-STL) with eoHM. The initial clinical records of these 17 patients did not show recognizable signs of other primary diseases except for high myopia, and further specific clinical examinations confirmed the diagnosis of IRDs or Stickler syndrome through eoHM. The PPI network analysis identified 87 candidate genes associated with early-onset high myopia (eoHM), grouped into four functional clusters. Thirteen hub genes, including RPGR, COL5A1, CRYAB, and FBN1, were crucial for the pathogenesis of myopia. The network showed strong biological relevance with highly significant enrichment (p-value < 1.0e-16). Our study expands the list of candidate genes associated with eoHM and suggests that eoHM may be the first reason for children to visit an ophthalmology clinic and an important clue for clinicians to detect underlying ocular diseases. These findings highlight the complex interplay of these genes, providing valuable insights into the molecular mechanisms of myopia and potential targets for future therapeutic interventions.
format Article
id doaj-art-126a43669090465b943a8942b60ef4b2
institution OA Journals
issn 2045-2322
language English
publishDate 2025-04-01
publisher Nature Portfolio
record_format Article
series Scientific Reports
spelling doaj-art-126a43669090465b943a8942b60ef4b22025-08-20T01:54:26ZengNature PortfolioScientific Reports2045-23222025-04-0115111510.1038/s41598-025-95574-xWhole-exome sequencing screening for candidate genes and potential pathogenic variants associated with early-onset high myopia in 47 Chinese familiesXue Rui0Huiping Li1Runqing Ma2Shangying Yang3Yuanyuan lian4Wanyu Cheng5Meijiao Ma6Weining Rong7Xunlun Sheng8Gansu Aier Ophthalmology and Optometry HospitalPeople’s Hospital of Ningxia Hui Autonomous Region, Ningxia Medical UniversityPeople’s Hospital of Ningxia Hui Autonomous Region, Ningxia Medical UniversityPeople’s Hospital of Ningxia Hui Autonomous Region, Ningxia Medical UniversityGansu Aier Ophthalmology and Optometry HospitalPeople’s Hospital of Ningxia Hui Autonomous Region, Ningxia Medical UniversityGansu Aier Ophthalmology and Optometry HospitalPeople’s Hospital of Ningxia Hui Autonomous Region, Ningxia Medical UniversityGansu Aier Ophthalmology and Optometry HospitalAbstract Early-onset high myopia (eoHM) occurs before school age and is an ideal model for monogenic studies of high myopia due to minimal environmental influence. This study screened for genes and variants associated with eoHM in 47 unrelated Chinese patients with eoHM. Protein–protein interaction (PPI) network analysis was conducted to detect interactions among candidate genes, and protein–protein docking was performed. In 28 patients (28/47, 59.6%), 32 potential pathogenic variants in 22 candidate genes were identified, including 24 novel variants. Among these 28 patients, 64.3% (18/28) carried pathogenic variants in RetNet genes. Of these, 12 patients (42.9%, 12/28) had pathogenic variants in six known genes (TSPAN12, CACNA1F, USH2A, RPGR, COL2A1, and COL11A1), which are responsible for retinal dystrophy and Stickler syndrome associated with eoHM. Additionally, 7 patients (25.0%, 7/28) carried pathogenic variants in seven candidate genes for ocular disease (POLA1, TMEM231, HK1, GSN, COL5A1, CRYBB3, and WDR), which were identified as potentially pathogenic in Chinese eoHM patients for the first time. Phenotype analysis showed that 11 patients presented with only high myopia, 10 patients had inherited retinal diseases (IRDs) with eoHM, and 7 patients had ocular-only Stickler syndrome (Ocular-STL) with eoHM. The initial clinical records of these 17 patients did not show recognizable signs of other primary diseases except for high myopia, and further specific clinical examinations confirmed the diagnosis of IRDs or Stickler syndrome through eoHM. The PPI network analysis identified 87 candidate genes associated with early-onset high myopia (eoHM), grouped into four functional clusters. Thirteen hub genes, including RPGR, COL5A1, CRYAB, and FBN1, were crucial for the pathogenesis of myopia. The network showed strong biological relevance with highly significant enrichment (p-value < 1.0e-16). Our study expands the list of candidate genes associated with eoHM and suggests that eoHM may be the first reason for children to visit an ophthalmology clinic and an important clue for clinicians to detect underlying ocular diseases. These findings highlight the complex interplay of these genes, providing valuable insights into the molecular mechanisms of myopia and potential targets for future therapeutic interventions.https://doi.org/10.1038/s41598-025-95574-x
spellingShingle Xue Rui
Huiping Li
Runqing Ma
Shangying Yang
Yuanyuan lian
Wanyu Cheng
Meijiao Ma
Weining Rong
Xunlun Sheng
Whole-exome sequencing screening for candidate genes and potential pathogenic variants associated with early-onset high myopia in 47 Chinese families
Scientific Reports
title Whole-exome sequencing screening for candidate genes and potential pathogenic variants associated with early-onset high myopia in 47 Chinese families
title_full Whole-exome sequencing screening for candidate genes and potential pathogenic variants associated with early-onset high myopia in 47 Chinese families
title_fullStr Whole-exome sequencing screening for candidate genes and potential pathogenic variants associated with early-onset high myopia in 47 Chinese families
title_full_unstemmed Whole-exome sequencing screening for candidate genes and potential pathogenic variants associated with early-onset high myopia in 47 Chinese families
title_short Whole-exome sequencing screening for candidate genes and potential pathogenic variants associated with early-onset high myopia in 47 Chinese families
title_sort whole exome sequencing screening for candidate genes and potential pathogenic variants associated with early onset high myopia in 47 chinese families
url https://doi.org/10.1038/s41598-025-95574-x
work_keys_str_mv AT xuerui wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies
AT huipingli wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies
AT runqingma wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies
AT shangyingyang wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies
AT yuanyuanlian wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies
AT wanyucheng wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies
AT meijiaoma wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies
AT weiningrong wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies
AT xunlunsheng wholeexomesequencingscreeningforcandidategenesandpotentialpathogenicvariantsassociatedwithearlyonsethighmyopiain47chinesefamilies