Exonic Knockout and Knockin Gene Editing in Hematopoietic Stem and Progenitor Cells Rescues RAG1 Immunodeficiency.
Maria Carmina Castiello,Chiara Brandas,Samuele Ferrari,Simona Porcellini,Nicolo Sacchetti,Daniele Canarutto,Elena Draghici,Ivan Merelli,Matteo Barcella, Gabriele Pelosi,Valentina Vavassori,Angelica Varesi,Aurelien Jacob,Serena Scala,Luca Basso Ricci,Marianna Paulis,Dario Strina,Martina Di Verniere, Sergi,Marta Serafini,Steven M. Holland,Jenna R. E. Bergerson,Suk See De Ravin,Harry L. Malech,Francesca Pala,Marita Bosticardo,Chiara Brombin,Federica Cugnata,Enrica Calzoni,Gay M. Crooks,Luigi D. Notarangelo,Pietro Genovese,Luigi Naldini,Anna Villa Science Translational Medicine（2024）
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