ImmunologybioRxiv
Genetically Corrected RAG2-SCID Human Hematopoietic Stem CellsRestore V(D)J-Recombinase and Rescue Lymphoid Deficiency
M. Pavel-Dinu, C. L. Gardner, Y. Nakauchi…·M. H. Porteus
This study corrects the RAG2 gene in human hematopoietic stem cells to restore V(D)J recombination and lymphoid development in RAG2-SCID patients.