Article

Disruption of a six-nucleotide miRNA motif improves <i>PKD1</i> dosage and ameliorates polycystic kidney disease

2025-10-23

Abstract excerpt

<h4>ABSTRACT</h4> Disrupting microRNA interactions to restore protein expression from haploinsufficient genes offers a promising precision-therapy strategy for monogenic disorders. PKD1 heterozygosity underlies autosomal dominant polycystic kidney disease (ADPKD), a disorder affecting nearly 12 million people worldwide, where reduced PKD1 dosage drives progressive cyst formation and kidney failure. We previousl...

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Literature Corpus work: 122472a4-1d33-5d0a-861a-45cc016f866c
DOI: 10.1101/2025.10.22.683929

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