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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: hernandez gonzalez em. Blood Adv. 2024 Apr 9;8(7):1820-1833. doi: 10.1182/bloodadvances.2023011766. Blood Adv. 2024. PMID: 38096800 Free PMC article.