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CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.
Bortolussi, Giulia; Iaconcig, Alessandra; Canarutto, Giulia; Porro, Fabiola; Ferrucci, Filippo; Galletta, Claudia; Díaz-Muñoz, Cristian; Rawat, Vipin; De Caneva, Alessia; Olajide, Olayemi Joseph; Zentilin, Lorena; Piazza, Silvano; Bockor, Luka; Muro, Andrés Fernando.
Afiliação
  • Bortolussi G; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Iaconcig A; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Canarutto G; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Porro F; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Ferrucci F; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Galletta C; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Díaz-Muñoz C; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Rawat V; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • De Caneva A; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Olajide OJ; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Zentilin L; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Piazza S; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Bockor L; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Muro AF; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
Mol Ther Methods Clin Dev ; 31: 101161, 2023 Dec 14.
Article em En | MEDLINE | ID: mdl-38094199

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Itália

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Itália