Background: Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are neurodegenerative disorders with overlapping clinical, pathological and genetic features. Genetic factors are the only proven causes, yet identified variants explain disease in only ~15% of ALS and ~30% of FTD patients. Traditional gene discovery approaches, like linkage analysis for highly penetrant families and genome-wide association studies in sporadic cohort, have limited power in smaller or reduced-penetrance cohorts. Alternative strategies are needed to bridge the gap.
We use identity-by-descent (IBD) analysis as an alternative method for gene discovery in an integrated cohort of ALS and FTD cases. We hypothesis that IBD can uncover previously unknown relatives among affected individuals, enabling pedigree reconstruction and identification of shared inherited genomic regions for downstream variant discovery using whole-genome sequencing (WGS).
Methods: Pairwise IBD analysis was performed using XIBD (1) on SNP genotype data from ~3,500 ALS and FTD patients of European ancestry from Australia, New Zealand and the United Kingdom. Relationship degrees were estimated using KING (2). For one IBD-linked pedigree, short-read WGS was interrogated for SNVs, indels and repeat expansions (3,4) within shared IBD regions. Variants were validated using IGV (5) and REViewer (6).
Results: 793 pairs of 1st-6th degree relatives were identified, one-third of which were previously unknown, including ~5% of sporadic ALS and ~16% of sporadic FTD cases. We identified 76 IBD-linked pedigrees for gene discovery. Comparative genomics of one IBD pedigree revealed candidate variants within a shared locus (LOD = 3.3).
Conclusions: IBD inference enables pedigree reconstruction and variant discovery across heterogeneous ALS and FTD cohorts, providing a scalable strategy to uncover missing heritability beyond traditional methods.