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Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice.
Lisjak, Michela; De Caneva, Alessia; Marais, Thibaut; Barbon, Elena; Biferi, Maria Grazia; Porro, Fabiola; Barzel, Adi; Zentilin, Lorena; Kay, Mark A; Mingozzi, Federico; Muro, Andrés F.
Afiliação
  • Lisjak M; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • De Caneva A; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Marais T; Inserm UMRS974, Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Université, Paris, France.
  • Barbon E; Genethon, Evry, France.
  • Biferi MG; IRCCS San Raffaele Hospital, Milan, Italy.
  • Porro F; Inserm UMRS974, Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Université, Paris, France.
  • Barzel A; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Zentilin L; Department of Biochemistry and Molecular Biology, The George S. Wise Faculty of Life Sciences, Tel Aviv University, Tel Aviv, Israel.
  • Kay MA; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
  • Mingozzi F; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA, United States.
  • Muro AF; Genethon, Evry, France.
Front Genome Ed ; 4: 785698, 2022.
Article em En | MEDLINE | ID: mdl-35359664

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Ano de publicação: 2022 Tipo de documento: Article