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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Mol Ther
Egile Nagusiak: 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.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Gene & Cell Therapy 2021
Gaiak:
Original Article
Sarrera elektronikoa: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
Etiketak: Etiketa erantsi
Etiketarik gabe, Izan zaitez lehena erregistro honi etiketa jartzen!

Antzeko izenburuak