Collecting Duct-Targeted Lipid Nanoparticles Deliver <i>Pkd2</i> mRNA to Restore Polycystin-2 and Attenuate ADPKD

Abstract excerpt

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 rath...

Topics

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• Body Fluids
• Chlorides
• Extracellular Matrix
• PKD1 gene
• Kidney Tubules
• Mutation
• Polycystic Kidney, Autosomal Dominant
• Proteins

Identifiers and source

Literature Corpus work

940683d1-e0ac-5cdd-a4b5-8e93efe102bd

DOI

10.64898/2026.06.02.728835