Article

CRISPR activation of endogenous <i>PKD1</i> increases polycystin-1 levels and suppresses cellular features of ADPKD

2026-05-25

Abstract excerpt

Most cases of autosomal dominant polycystic kidney disease (ADPKD) are caused by mutations in PKD1, which reduce polycystin-1 (PC1) levels below a critical functional threshold. Normalizing PC1 dosage mitigates disease progression; therefore, we sought to develop a CRISPR activation (CRISPRa) strategy to transcriptionally upregulate endogenous PKD1 . We systematically screened multiple single-guide RNAs using an...

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Identifiers and source

Literature Corpus work: 15bb778f-bf53-5239-b893-8df851056a95
DOI: 10.64898/2026.05.23.727418

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