Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

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 :: Blood Advances; 20240101, Issue: Preprints
Publication Year :: 2024
Abstract: •Human hematopoietic stem cells can be corrected to restore endogenous RAG2gene expression while preserving durable engraftment potential.•Gene-corrected RAG2locus restores V(D)J recombination in stem cells of patients with RAG2-SCID, promoting TCR and B-cell receptor formation.

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