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

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...

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Bibliografiset tiedot
Julkaisussa:Mol Ther
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Gene & Cell Therapy 2021
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Original Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC8116578/
https://ncbi.nlm.nih.gov/pubmed/33484963
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2021.01.024
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