Correction of a urea cycle defect after ex vivo gene editing of human hepatocytes.

doi:10.1016/j.ymthe.2021.01.024

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Article

Correction of a urea cycle defect after ex vivo gene editing of human hepatocytes.

Molecular therapy : the journal of the American Society of Gene Therapy - 5 May 2021

Zabulica Mihaela, Srinivasan Raghuraman C, Akcakaya Pinar, Allegri Gabriella, Bestas Burcu, Firth Mike, Hammarstedt Christina, Jakobsson Tomas, Jakobsson Towe, Ellis Ewa, Jorns Carl, Makris Georgios, Scherer Tanja, Rimann Nicole, van Zuydam Natalie R, Gramignoli Roberto, Forslöw Anna, Engberg Susanna, Maresca Marcello, Rooyackers Olav, Thöny Beat, Häberle Johannes, Rosen Barry, Strom Stephen C

Abstract excerpt

Ornithine transcarbamylase deficiency (OTCD) is a monogenic disease of ammonia metabolism in hepatocytes. Severe disease is frequently treated by orthotopic liver transplantation. An attractive approach is the correction of a patient's own cells to regenerate the liver with gene-repaired hepatocytes. This study investigates the efficacy and safety of ex vivo correction of primary human hepatocytes. Hepatocytes...

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Identifiers

PMID: 33484963
DOI: 10.1016/j.ymthe.2021.01.024
PMCID: PMC8116578

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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