Polycystic kidney disease is the most common monogenic cause of kidney failure and affects over 10 million individuals. We've known most PKD is caused by mutations in PKD1 for over 30 years. But how PKD1 prevents disease has remained mysterious, limiting effective therapy.
🧬 In #ADPKD, reduced PKD1 drives cyst growth and kidney failure.
We previously discovered a 55bp cis-repressive element in its 3'UTR.
We now identify the six critical bases—the miR-17 motif—whose disruption or masking restores PKD1 and slows disease.
🔗 https://t.co/hYIveEkPPU
Check out our latest manuscript where we generate a cross model single cell and spatial atlas of mouse PKD!
A cross model spatial and single-cell atlas reveals the conserved involvement of osteopontin in polycystic kidney disease | bioRxiv https://t.co/ICBC4SXIXJ
Thrilled to share our work and truly honored to share senior autorship with @FabiolaTerzi ! Thanks @ScienceTM for publishing our work ! https://t.co/hV96gO39Mq