Ontology highlight
ABSTRACT:
SUBMITTER: Famulari ES
PROVIDER: S-EPMC6972964 | biostudies-literature | 2020 Jan
REPOSITORIES: biostudies-literature
Famulari Elvira Smeralda ES Navarro-Tableros Victor V Herrera Sanchez Maria Beatriz MB Bortolussi Giulia G Gai Marta M Conti Laura L Silengo Lorenzo L Tolosano Emanuela E Tetta Ciro C Muro Andrés Fernando AF Camussi Giovanni G Fagoonee Sharmila S Altruda Fiorella F
Scientific reports 20200121 1
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 SCID Gamma (NSG)/Ugt1<sup>-/-</sup> mice, which closely mimic the pathological manife ...[more]