Functional interrogation of twenty type 2 diabetes-associated genes using isogenic human embryonic stem cell-derived β-like cells

Cell Metab. 2023 Nov 7;35(11):1897-1914.e11. doi: 10.1016/j.cmet.2023.09.013. Epub 2023 Oct 18.

Abstract

Genetic studies have identified numerous loci associated with type 2 diabetes (T2D), but the functional roles of many loci remain unexplored. Here, we engineered isogenic knockout human embryonic stem cell lines for 20 genes associated with T2D risk. We examined the impacts of each knockout on β cell differentiation, functions, and survival. We generated gene expression and chromatin accessibility profiles on β cells derived from each knockout line. Analyses of T2D-association signals overlapping HNF4A-dependent ATAC peaks identified a likely causal variant at the FAIM2 T2D-association signal. Additionally, the integrative association analyses identified four genes (CP, RNASE1, PCSK1N, and GSTA2) associated with insulin production, and two genes (TAGLN3 and DHRS2) associated with β cell sensitivity to lipotoxicity. Finally, we leveraged deep ATAC-seq read coverage to assess allele-specific imbalance at variants heterozygous in the parental line and identified a single likely functional variant at each of 23 T2D-association signals.

Keywords: HNF4A; allelic imbalance; cellular trait; differentiation; genome-wide association studies; insulin production; lipotoxicity; single nucleotide polymorphism; β cells.

Publication types

  • Research Support, N.I.H., Intramural
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Carbonyl Reductase (NADPH) / genetics
  • Carbonyl Reductase (NADPH) / metabolism
  • Diabetes Mellitus, Type 2* / genetics
  • Diabetes Mellitus, Type 2* / metabolism
  • Genetic Predisposition to Disease
  • Genome-Wide Association Study
  • Human Embryonic Stem Cells* / metabolism
  • Humans
  • Insulin-Secreting Cells* / metabolism
  • Polymorphism, Single Nucleotide

Substances

  • DHRS2 protein, human
  • Carbonyl Reductase (NADPH)