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Article

In vivo base editing rescues ADPKD in a humanized mouse model.

Nature communications - 11 Dec 2025

Cheng Alice Shasha, Li Linda Xiaoyan, Zhou Julie Xia, Harris Peter C, Calvet James P, Li Xiaogang

Abstract excerpt

Autosomal dominant polycystic kidney disease (ADPKD) is a genetic kidney disease, caused by mutations of the PKD1 and PKD2 genes, characterized by the development of renal cysts and extrarenal complications, such as cardiac hypertrophy. Recently, a revolutionary approach, adeno-associated virus (AAV) delivered CRISPR-Cas9 gene editing, has been developed to treat inherited diseases. However, the use of this...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 41381416
DOI: 10.1038/s41467-025-65997-1
PMCID: PMC12714704

Genes

PKD1
PKD2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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