GeneticsMolecular TherapyPeer-reviewed
In vivo base editing reduces liver cysts in autosomal dominant polycystic kidney disease
Antonia Ibel, Rishi Bhardwaj, Duygu Elif Yılmaz…·Michael M. Kaminski
This study shows that CRISPR base editing can correct pathogenic PKD1 variants in mice, reducing liver cysts and increasing polycystin-1 expression.
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