ADPKD
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Related Articles from SNS
Collecting Duct-Targeted Lipid Nanoparticles Deliver Pkd2 mRNA to Restore Polycystin-2 and Attenuate ADPKD
Autosomal dominant polycystic kidney disease (ADPKD), a leading genetic cause of kidney failure, is caused by mutations in the PKD1 or PKD2 genes, resulting in functional polycystin 1 (PC1) or polycystin 2 (PC2) deficiency, and is characterized by progressive cyst expansion in the kidneys. However, no approved therapy directly restores polycystin expression, and current treatments target downstream pathways rather than the genetic defect. Gene replacement therapy offers a direct route to...