Gene Correction for SCID-X1 in Long-Term Hematopoietic Stem Cells
M. Pavel-Dinu, V. Wiebking, B. T. Dejene, W. Srifa, S. Mantri, C. Nicolas, C. Lee, G. Bao, E. J. Kildebeck, N. Punjya, C. Sindhu, M. A. Inlay, N. Saxena, S. S. DeRavin, H. Malech, , 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.
bioRxiv · Aug 22, 2018Bioengineering