Federal Project Grant Award Summary

Children's Research Institute received a $156,814 Project Grant from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847), effective August 1, 2026, with a completion date of May 31, 2031. The award funds investigator-initiated basic biomedical research to advance understanding of autosomal recessive polycystic kidney disease (ARPKD), a severe fibrocystic disorder affecting the kidneys and liver in approximately 120 neonates annually in the United States.

The research project delivers mechanistic studies defining immunomodulatory pathways in ARPKD pathogenesis using a novel double knockout mouse model, human kidney tissue, and genomic datasets. The research program comprises three primary aims: (1) characterizing the timing and location of kidney cyst initiation and immune cell infiltration; (2) employing single-cell RNA sequencing to identify immune-mediated transcriptomic alterations driving disease progression, integrated with human ARPKD kidney datasets; and (3) determining the functional role of the PKHD1L1 protein in epithelial and immune signaling. The research is intended to identify novel therapeutic targets and advance targeted treatment options for ARPKD, for which no disease-modifying therapies currently exist.