Congenital and Acute Kidney Disease: Translational Research Insights from Zebrafish Chemical Genetics

Today, acute kidney injury (AKI) and congenital anomalies of the kidney and urinary tract (CAKUT) represent major issues in healthcare. Both AKI and CAKUT can lead to end stage renal disease (ESRD) that requires life-long medical care with renal replacement therapy. Renal replacement by dialysis is intensive, and kidney transplantation is restricted by organ availability. These limitations, along with the growing epidemic of patients affected by kidney disease, highlight the significant need to identify alternative ways to treat renal injury and birth defects. Drug discovery is one promising avenue of current research. Here, we discuss zebrafish chemical genetics and its latent potency as a method to rapidly identify small molecule therapeutics to accelerate recovery after AKI. Specifically, we review two groundbreaking studies that have recently provided a template to screen for compounds that expand the renal progenitor field in development that were capable of treating AKI in both the zebrafish and the mouse. These new findings demonstrate that drug discovery using zebrafish can be used for relevant translational research to identify clinical interventions for renal conditions in humans.