ArticleAdvanced biotechnology2024
Adenine base editor corrected ADPKD point mutations in hiPSCs and kidney organoids.
Article in Advanced biotechnology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed.
- Review
- Gene modification: Exploring the potential in treating kidney diseases.Pharmacological research · 2026Review
- Review
- Kidney Organoids: Current Advances and Applications.Life (Basel, Switzerland) · 2025Review
- CRISPR and gene editing for kidney diseases: where are we?Clinical kidney journal · 2025Review
- Advances and Challenges in Modeling Autosomal Dominant Polycystic Kidney Disease: A Focus on Kidney Organoids.Biomedicines · 2025Review
- Prospects for gene therapy in polycystic kidney disease.Current opinion in nephrology and hypertension · 2025Review
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Authors and funding
12 authors.
Funding
Abstract
Autosomal dominant polycystic kidney disease (ADPKD) is a dominant genetic disorder caused primarily by mutations in the PKD1 gene, resulting in the formation of numerous cysts and eventually kidney failure. However, there are currently no gene therapy studies aimed at correcting PKD1 gene mutations. In this study, we identified two mutation sites associated with ADPKD, c.1198 (C > T) and c.8311 (G > A), which could potentially be corrected by adenine base editor (ABE). The correction efficiencies of different ABE variants were tested using the HEK293T-PKD1 c.1198 (C > T) and HEK293T-PKD1 c.8311 (G > A) reporter cell lines. We then generated induced pluripotent stem cells (iPSCs
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