BioengineeringbioRxiv
Gene Correction for SCID-X1 in Long-Term Hematopoietic Stem Cells
M. Pavel-Dinu, V. Wiebking, B. T. Dejene…·M. Porteus
This study corrects SCID-X1 in hematopoietic stem cells using CRISPR/Cas9, achieving high integration frequencies and low off-target effects, supporting clinical development.