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Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids

  • Lucia Nicosia
  • , Iwona Pranke
  • , Roberta V. Latorre
  • , Joss B. Murray
  • , Lisa Lonetti
  • , Kader Cavusoglu-Doran
  • , Elise Dreano
  • , James P. Costello
  • , Michael Carroll
  • , Paola Melotti
  • , Claudio Sorio
  • , Isabelle Sermet-Gaudelus
  • , Martina F. Scallan
  • , Patrick T. Harrison
  • Université Paris Descartes-Sorbonne-Paris Cité
  • Université Paris Cité
  • University of Verona
  • Azienda Ospedaliera Universitaria Integrata di Verona
  • European Reference Network
  • Cincinnati Children's Hospital Medical Center

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Neuroscience

Biochemistry, Genetics and Molecular Biology