Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency
- Resource Type
- Article
- Authors
- Pavel-Dinu, Mara; Gardner, Cameron L.; Nakauchi, Yusuke; Kawai, Tomoki; Delmonte, Ottavia M.; Palterer, Boaz; Bosticardo, Marita; Pala, Francesca; Viel, Sebastien; Malech, Harry L.; Ghanim, Hana Y.; Bode, Nicole M.; Kurgan, Gavin L.; Detweiler, Angela M.; Vakulskas, Christopher A.; Neff, Norma F.; Sheikali, Adam; Menezes, Sherah T.; Chrobok, Jade; Hernández González, Elaine M.; Majeti, Ravindra; Notarangelo, Luigi D.; Porteus, Matthew H.
- Source
- In Blood Advances 9 April 2024 8(7):1820-1833
- Subject
- Gene Therapy
- Language
- ISSN
- 2473-9529