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Article

Gene Editing Correction of a Urea Cycle Defect in Organoid Stem Cell Derived Hepatocyte-like Cells.

International journal of molecular sciences - 26 Jan 2021

Zabulica Mihaela, Jakobsson Tomas, Ravaioli Francesco, Vosough Massoud, Gramignoli Roberto, Ellis Ewa, Rooyackers Olav, Strom Stephen C

Abstract excerpt

Urea cycle disorders are enzymopathies resulting from inherited deficiencies in any genes of the cycle. In severe cases, currently available therapies are marginally effective, with liver transplantation being the only definitive treatment. Donor liver availability can limit even this therapy. Identification of novel therapeutics for genetic-based liver diseases requires models that provide measurable hepatic...

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Identifiers

PMID: 33530582
DOI: 10.3390/ijms22031217
PMCID: PMC7865883

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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