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

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

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. Ident...

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Bibliographic Details
Main Authors: Mihaela Zabulica, Tomas Jakobsson, Francesco Ravaioli, Massoud Vosough, Roberto Gramignoli, Ewa Ellis, Olav Rooyackers, Stephen C. Strom
Format: Article
Language:English
Published: MDPI AG 2021-01-01
Series:International Journal of Molecular Sciences
Subjects:
iPSC
hepatocytes
disease modelling
genome editing
CRISPR
urea cycle
Online Access:https://www.mdpi.com/1422-0067/22/3/1217

Internet

https://www.mdpi.com/1422-0067/22/3/1217

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