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Is there any disease in the liver that cannot be corrected with base editing?

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Base editing has shown promise in correcting genetic liver diseases. However, some severe cases, like urea cycle disorders, may still pose challenges. These enzymopathies result from inherited deficiencies in urea cycle genes, requiring liver transplantation as the definitive treatment due to limited donor availability . While base editing has been successful in correcting phenylketonuria in mice without off-target mutations, some liver disorders may still present complexities. Liver diseases involving defective or non-functional proteins have been targeted with gene therapy and genome editing, particularly using base editors, to correct DNA-level defects . Despite advancements, the effectiveness of base editing in addressing all liver diseases, especially those with complex genetic underpinnings, may vary, necessitating further research and tailored approaches.

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Open access•Journal Article•DOI Liver-specific in vivo base editing of Angptl3 via AAV delivery efficiently lowers blood lipid levels in mice Yuanbojiao Zuo, Chen Zhang, Haiwen Li, Weidong Xiao, Roland W. Herzog, Jie Xu, Jifeng Zhang, Y. Eugene Chen - Show less +7 more 15 Jun 2023-Cell & Bioscience	Not addressed in the paper.
Open access•Journal Article•DOI Genome editing technologies to treat rare liver diseases Marta Trevisan, Giulia Masi, Giorgio Palù - Show less +2 more 05 Apr 2020-Translational Gastroenterology and Hepatology 10 Citations	Base editing technologies have shown promise in correcting defects underlying rare liver diseases, suggesting potential for correcting a wide range of hepatic disorders.
Open access•Journal Article•DOI Gene Editing Correction of a Urea Cycle Defect in Organoid Stem Cell Derived Hepatocyte-like Cells. Mihaela Zabulica, Tomas Jakobsson, Francesco Ravaioli, Massoud Vosough, Roberto Gramignoli, Ewa Ellis, Olav Rooyackers, Stephen C. Strom - Show less +7 more 26 Jan 2021-International Journal of Molecular Sciences 13 Citations	Not addressed in the paper.
Journal Article•DOI Therapeutic base editing in the adult liver Coen C. Paulusma, Piter J. Bosma - Show less +1 more 01 Sep 2021-Nature Reviews Gastroenterology & Hepatology 1 Citations	Not addressed in the paper.
Open access•Journal Article•DOI In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA Lukas Villiger, Lukas Villiger, Tanja Rothgangl, Tanja Rothgangl, Dominik Witzigmann, Dominik Witzigmann, Rurika Oka, Paulo J.C. Lin, Weihong Qi, Sharan Janjuha, Sharan Janjuha, Christian Berk, Femke Ringnalda, Mitchell B. Beattie, Markus Stoffel, Beat Thöny, Beat Thöny, Jonathan Hall, Hubert Rehrauer, Ruben van Boxtel, Ying K. Tam, Gerald Schwank, Gerald Schwank - Show less +22 more 25 Jan 2021-Nature Biomedical Engineering 40 Citations	Not addressed in the paper.

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Related Questions

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