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Recessive genetic contribution to congenital heart disease in 5,424 probands.

Proceedings of the National Academy of Sciences of the United States of America2025-03-03PubMed
Total: 81.5Innovation: 9Impact: 8Rigor: 8Citation: 7

Summary

Whole-exome analysis of 5,424 CHD probands estimates that rare recessive genotypes explain at least 2.2% of CHD overall and 5.4% in laterality phenotypes, with clear enrichment in consanguinity. The study catalogs recessive hits across curated CHD genes, refining genetic counseling and research priorities.

Key Findings

  • Rare damaging recessive genotypes contribute to ≥2.2% of CHD overall and 5.4% in laterality phenotypes.
  • Among 108 curated recessive CHD genes, 66 recessive genotypes were identified; 11 genes harbored >1 recessive genotype.
  • Recessive genotypes were more prevalent in consanguineous offspring (4.7%) than in nonconsanguineous probands (0.7%).

Clinical Implications

Enhances risk counseling, especially in consanguineous populations and laterality defects; informs gene panel design and prioritization for molecular diagnosis; sets baselines for recessive disease burden in CHD.

Why It Matters

Provides the largest systematic quantification of recessive contribution across CHD phenotypes and identifies target genes/phenotypes for future discovery. This shifts understanding beyond anecdotal consanguineous families to population-level estimates.

Limitations

  • Abstract truncation suggests incomplete reporting of founder variants and full gene list in this extract.
  • Exome-based approach may miss non-coding/regulatory variants and structural variants.

Future Directions

Expand to whole-genome sequencing for regulatory variants; deepen analyses of laterality pathways; integrate functional validation to confirm causality and mechanisms.

Study Information

Study Type
Cohort
Research Domain
Pathophysiology
Evidence Level
II - Large genetic cohort study with systematic exome analysis and phenotype stratification
Study Design
OTHER