Alport syndrome articles from across Nature Portfolio

The development of a kidney organoid model from induced pluripotent stem cells (iPSCs) recapitulates collagen composition from mild and severe cases of Alport syndrome, establishing a kidney organoid model aiding drug discovery.

This study describes a class of small molecule compounds that promote ABCA1-dependent cholesterol efflux via a non-transcriptional mechanism, the identification of the molecular target by a chemical biology approach, and the potential of these agents for the treatment of chronic kidney diseases and potentially other diseases where lipid accumulation drives disease progression.

Proper glomerular basement membrane (GBM) structure and function are required for appropriate glomerular filtration and abnormalities in the α3α4α5 (α345) network of collagen IV — a GBM component — can lead to disease. New insights into the mechanisms underlying α345 hexamer assembly and function from genetic and structural data identify possible targets for therapeutic modulation.