Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency.
Pavel-Dinu M, Gardner CL, Nakauchi Y, Kawai T, Delmonte OM, Palterer B, Bosticardo M, Pala F, Viel S, Malech HL, Ghanim HY, Bode NM, Kurgan GL, Detweiler AM, Vakulskas CA, Neff NF, Sheikali A, Menezes ST, Chrobok J, Hernández González EM, Majeti R, Notarangelo LD, Porteus MH.
Pavel-Dinu M, et al. Among authors: nakauchi y.
Blood Adv. 2024 Apr 9;8(7):1820-1833. doi: 10.1182/bloodadvances.2023011766.
Blood Adv. 2024.
PMID: 38096800
Free PMC article.