Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency
造血干细胞和祖细胞中的外显子敲除和敲入基因编辑可挽救 RAG1 免疫缺陷
期刊:Science Translational Medicine
影响因子:15.8
doi:10.1126/scitranslmed.adh8162
Maria Carmina Castiello, Chiara Brandas, Samuele Ferrari, Simona Porcellini, Nicolò 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, Marti