Functional properties of the R154X HNF‐4α protein generated by a mutation associated with maturity‐onset diabetes of the young, type 1

Abstract
Mutations in the hepatocyte nuclear factor 4α (HNF-4α) gene are associated with one form of maturity-onset diabetes of the young (MODY1). The R154X mutation generates a protein lacking the E-domain which is required for normal HNF-4α functions. Since pancreatic β-cell dysfunction is a feature of MODY1 patients, we compared the functional properties of the R154X mutant in insulin-secreting pancreatic β-cells and non-β-cells. The R154X mutation did not affect nuclear localisation in β-cells and non-β-cells. However, it did lead to a greater impairment of HNF-4α function in β-cells compared to non-β-cells, including a complete loss of transactivation activity and a dominant-negative behaviour.

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