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Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T-cells for the treatment of Hyper IgM1.
Canarutto, Daniele; Asperti, Claudia; Vavassori, Valentina; Porcellini, Simona; Rovelli, Elisabetta; Paulis, Marianna; Ferrari, Samuele; Varesi, Angelica; Fiumara, Martina; Jacob, Aurelien; Sergi Sergi, Lucia; Visigalli, Ilaria; Ferrua, Francesca; González-Granado, Luis Ignacio; Lougaris, Vassilios; Finocchi, Andrea; Villa, Anna; Radrizzani, Marina; Naldini, Luigi.
Affiliation
  • Canarutto D; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Asperti C; Università Vita-Salute San Raffaele, Milan, Italy.
  • Vavassori V; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Porcellini S; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Rovelli E; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Paulis M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Ferrari S; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Varesi A; Humanitas Clinical and Research Center IRCCS, Milan, Italy.
  • Fiumara M; UOS Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), CNR, Milan, Italy.
  • Jacob A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Sergi Sergi L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Visigalli I; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Ferrua F; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • González-Granado LI; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Lougaris V; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Finocchi A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Villa A; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Radrizzani M; Unidad de Immunodeficiencias Primarias y la Unidad de Hematología y Oncología Pediátrica, Instituto de Investigacíon Hospital 12 de Octubre, Madrid, Spain.
  • Naldini L; University of Brescia, Brescia, Italy.
EMBO J ; 42(23): e114188, 2023 Dec 01.
Article in En | MEDLINE | ID: mdl-37916874
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: CRISPR-Cas Systems / Gene Editing Limits: Humans Language: En Journal: EMBO J Year: 2023 Type: Article Affiliation country: Italy
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: CRISPR-Cas Systems / Gene Editing Limits: Humans Language: En Journal: EMBO J Year: 2023 Type: Article Affiliation country: Italy