Inhibiting GLIS2 for autosomal dominant polycystic kidney disease

Inhibiting the GLI-similar zinc finger transcription factor GLIS2 could help treat autosomal dominant polycystic kidney disease (ADPKD), which can also be caused by homozygous recessive loss of function mutations in the cation channel regulator PKD1, by preventing cyst formation in the kidneys.

Sequencing of ribosome-associated RNA transcripts, or translatomes, from mouse kidney tubule cells identified higher GLIS2 expression in mice with ADPKD induced by homozygous renal tubular epithelial cell-specific PKD1 knockout than in non-cystic mice heterozygous for PKD1, or homozygous knockout mice protected from cyst formation by cell-specific knockout of the cilia regulator KIF3A. ...