Human liver stem cells express UGT1A1 and improve phenotype of immunocompromised Crigler Najjar syndrome type I mice

Crigler Najjar Syndrome type I (CNSI) is a rare recessive disorder caused by mutations in the Ugt1a1 gene. There is no permanent cure except for liver transplantation, and current therapies present several shortcomings. Since stem cell-based therapy offers a promising alternative for the treatment o...

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Udgivet i:Sci Rep
Main Authors: Famulari, Elvira Smeralda, Navarro-Tableros, Victor, Herrera Sanchez, Maria Beatriz, Bortolussi, Giulia, Gai, Marta, Conti, Laura, Silengo, Lorenzo, Tolosano, Emanuela, Tetta, Ciro, Muro, Andrés Fernando, Camussi, Giovanni, Fagoonee, Sharmila, Altruda, Fiorella
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2020
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6972964/
https://ncbi.nlm.nih.gov/pubmed/31965023
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-020-57820-2
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