Genetically Corrected RAG2-SCID Human Hematopoietic Stem CellsRestore V(D)J-Recombinase and Rescue Lymphoid Deficiency
M. Pavel-Dinu, C. L. Gardner, Y. Nakauchi, T. Kawai, O. M. Delmonte, B. Palterer, M. Bosticardo, F. Pala, S. Viel, H. L. Malech, H. Y. Ghanim, N. N. Bode, G. L. Kurgan, C. A. Vakulskas, A. Sheikali, , 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.
bioRxiv · Jul 12, 2022Immunology