Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
- Resource Type
- Article
- Authors
- Castiello, Maria Carmina; Brandas, Chiara; Ferrari, Samuele; Porcellini, Simona; Sacchetti, Nicolò; Canarutto, Daniele; Draghici, Elena; Merelli, Ivan; Barcella, Matteo; Pelosi, Gabriele; Vavassori, Valentina; Varesi, Angelica; Jacob, Aurelien; Scala, Serena; Basso Ricci, Luca; Paulis, Marianna; Strina, Dario; Di Verniere, Martina; Sergi Sergi, Lucia; Serafini, Marta
- Source
- Science Translational Medicine; 2/7/2024, Vol. 16 Issue 733, p1-18, 18p
- Subject
- SEVERE combined immunodeficiency
HEMATOPOIETIC stem cells
GENOME editing
GENE knockout
GENE expression
CYTOLOGY
- Language
- ISSN
- 19466234