Article
Human liver stem cells express UGT1A1 and improve phenotype of immunocompromised Crigler Najjar syndrome type I mice.
Scientific reports - 21 Jan 2020
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
Abstract excerpt
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 of this disorder, we evaluated the therapeutic potential of human liver stem cells (HLSC) in immune-compromised NOD...
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