Genetic and pharmacologic inhibition of ALDH1A3 as a treatment of β-cell failure. Author Jinsook Son, Wen Du, Mark Esposito, Kaavian Shariati, Hongxu Ding, Yibin Kang, Domenico Accili Publication Year 2023 Type Journal Article Abstract Type 2 diabetes (T2D) is associated with β-cell dedifferentiation. Aldehyde dehydrogenase 1 isoform A3 (ALHD1A3) is a marker of β-cell dedifferentiation and correlates with T2D progression. However, it is unknown whether ALDH1A3 activity contributes to β-cell failure, and whether the decrease of ALDH1A3-positive β-cells (A+) following pair-feeding of diabetic animals is due to β-cell restoration. To tackle these questions, we (i) investigated the fate of A+ cells during pair-feeding by lineage-tracing, (ii) somatically ablated ALDH1A3 in diabetic β-cells, and (iii) used a novel selective ALDH1A3 inhibitor to treat diabetes. Lineage tracing and functional characterization show that A+ cells can be reconverted to functional, mature β-cells. Genetic or pharmacological inhibition of ALDH1A3 in diabetic mice lowers glycemia and increases insulin secretion. Characterization of β-cells following ALDH1A3 inhibition shows reactivation of differentiation as well as regeneration pathways. We conclude that ALDH1A3 inhibition offers a therapeutic strategy against β-cell dysfunction in diabetes. Keywords Animals, Mice, Aldehyde Oxidoreductases, Cell Line, Tumor, Diabetes Mellitus, Type 2, Aldehyde Dehydrogenase 1 Family, Diabetes Mellitus, Experimental, Insulin-Secreting Cells Journal Nat Commun Volume 14 Issue 1 Pages 558 Date Published 2023/02/02 ISSN Number 2041-1723 DOI 10.1038/s41467-023-36315-4 Alternate Journal Nat Commun PMCID PMC9895451 PMID 36732513 PubMedPubMed CentralGoogle ScholarBibTeXEndNote X3 XML