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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: ghanim hy. Blood Adv. 2024 Apr 9;8(7):1820-1833. doi: 10.1182/bloodadvances.2023011766. Blood Adv. 2024. PMID: 38096800 Free PMC article.
Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic Antibodies.
Feist WN, Luna SE, Ben-Efraim K, Filsinger Interrante MV, Amorin NA, Johnston NM, Bruun TUJ, Ghanim HY, Lesch BJ, Dudek AM, Porteus MH. Feist WN, et al. Among authors: ghanim hy. bioRxiv [Preprint]. 2024 Mar 9:2024.03.08.583956. doi: 10.1101/2024.03.08.583956. bioRxiv. 2024. PMID: 38496600 Free PMC article. Preprint.