Open access
Sep 2026
CRISPR activation of endogenous PKD1 increases polycystin-1 levels and suppresses cellular features of ADPKD.
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 transcriptiona...
Anubhav Chakraborty, Meekha M. Varghese, Darren P. Wallace et al.
· AJP - Renal Physiology · 0 citations