Clonal haematopoiesis (CH) is an age-related process characterised by the accumulation of somatic mutations in haematopoietic stem cells (HSCs) that drive clonal expansion. CH is associated with increased risks of haematological malignancies and cardiovascular disease, yet its cellular and regulatory mechanisms remain unclear.
Here, as part of the TenK10K Phase 1 project, we generated a population scale multiomic resource comprising whole genome sequencing, genotyping arrays, single cell RNA sequencing, and single cell ATAC sequencing from more than 2,000 individuals and over 5 million peripheral blood mononuclear cells. This enabled systematic identification of diverse forms of CH at both the sample and single cell level.
We integrated summary statistics from CH genome-wide association studies with single cell transcriptomic profiles to map genetic risk to specific immune cell types. Using cell-type specific expression and chromatin accessibility QTL data, combined with colocalisation and causal inference, we identified CH associated genes and pathways and prioritised putative causal variants supported by fine mapping across multiple molecular layers. This generated the first catalogue of cell-type specific causal effects of gene expression on CH-related traits, yielding 4,515 associations across 260 genes and 28 cell types.
Leveraging matched scRNA-seq and scATAC-seq data, we characterised loss of the Y chromosome (LOY) across 974 male individuals. LOY was consistently enriched in monocytes, regulatory T cells, HSCs, and dendritic cells. Trajectory and expression analyses suggested that LOY may alter immune cell differentiation, including promoting the transition from naive CD4 T cells to regulatory T cells. This appears to be driven by increased FOXP3 expression that arises from compensatory upregulation of X-linked genes.
Together, this work provides a comprehensive multiomic framework for studying somatic variation and demonstrates how population scale single cell data can reveal the cellular contexts and regulatory mechanisms through which CH contributes to disease risk.