PUBLICATION

Genetic regulation of ARID3B confers cleft lip with/without cleft palate susceptibility through LLPS-mediated transcriptional program

Authors
Li, X., Li, D., Lou, S., Lin, J., Gao, Y., Vona, B., Mi, C., Wang, L., Ma, L., Du, M., Pan, Y.
ID
ZDB-PUB-251002-4
Date
2025
Source
Cell Reports   44: 116377116377 (Journal)
Registered Authors
Keywords
CP: Genomics, GWAS, cleft lip and cleft palate, genetics regulation, phase separation
MeSH Terms
  • Animals
  • Cell Movement/genetics
  • Cleft Lip*/genetics
  • Cleft Palate*/genetics
  • DNA-Binding Proteins*/genetics
  • DNA-Binding Proteins*/metabolism
  • Genetic Predisposition to Disease*
  • Humans
  • Polymorphism, Single Nucleotide/genetics
  • Transcription Factors*/genetics
  • Transcription Factors*/metabolism
  • Transcription, Genetic*
  • Zebrafish/genetics
PubMed
41032419 Full text @ Cell Rep.
Abstract
Genome-wide association studies have identified numerous loci associated with nonsyndromic cleft lip with/without cleft palate (nsCL/P). However, the causal genes within these loci remain to be systematically investigated. Through a multiomics screening strategy, we identified 20 candidate genes, with ARID3B emerging as the most significant risk gene for nsCL/P. Focusing on the ARID3B locus, we found a casual variant at rs1821848 that diminished the binding affinity of NR2C2, resulting in the upregulation of ARID3B. Notably, we observed the formation of ARID3B granules through liquid-liquid phase separation (LLPS) both in vivo and in vitro. This ARID3B-mediated LLPS could essentially recruit coactivators SMAD2/3 and establish enhancer activity necessary for initiating the gene profile related to nsCL/P. Ultimately, disrupting the LLPS of arid3b effectively rescued migration, apoptosis, and phenotype deficits in zebrafish models. This study systematically revealed biological functions of both rs1821848 and ARID3B during nsCL/P development.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping