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Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
Brusson, Mégane; Chalumeau, Anne; Martinucci, Pierre; Romano, Oriana; Felix, Tristan; Poletti, Valentina; Scaramuzza, Samantha; Ramadier, Sophie; Masson, Cecile; Ferrari, Giuliana; Mavilio, Fulvio; Cavazzana, Marina; Amendola, Mario; Miccio, Annarita.
Afiliación
  • Brusson M; Université de Paris, Imagine Institute, Laboratory of Chromatin and Gene Regulation During Development, INSERM UMR1163, 75015 Paris, France.
  • Chalumeau A; Université de Paris, Imagine Institute, Laboratory of Chromatin and Gene Regulation During Development, INSERM UMR1163, 75015 Paris, France.
  • Martinucci P; Université de Paris, Imagine Institute, Laboratory of Chromatin and Gene Regulation During Development, INSERM UMR1163, 75015 Paris, France.
  • Romano O; Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.
  • Felix T; Department of Molecular Medicine, University of Padova, 35131 Padova, Italy.
  • Poletti V; Université de Paris, Imagine Institute, Laboratory of Chromatin and Gene Regulation During Development, INSERM UMR1163, 75015 Paris, France.
  • Scaramuzza S; Woman and Child Health Department, University of Padova, Padova, Italy.
  • Ramadier S; Harvard Medical School, Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
  • Masson C; Pediatric Research Institute, City of Hope, Padova, Italy.
  • Ferrari G; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Mavilio F; Université de Paris, Imagine Institute, Laboratory of Chromatin and Gene Regulation During Development, INSERM UMR1163, 75015 Paris, France.
  • Cavazzana M; Paris-Descartes Bioinformatics Platform, Imagine Institute, 75015 Paris, France.
  • Amendola M; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Miccio A; Vita-Salute, San Raffaele University, Milan, Italy.
Mol Ther Nucleic Acids ; 32: 229-246, 2023 Jun 13.
Article en En | MEDLINE | ID: mdl-37090420

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2023 Tipo del documento: Article País de afiliación: Francia

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2023 Tipo del documento: Article País de afiliación: Francia