Reverse genetics in humanized mice reveals CARD8-mediated pyroptosis causing pancytopenia in human DPP9 deficiency
The results reveal a unique human mechanism of disease and offer therapeutic insight for this inflammasomopathy and CRISPR editing of human CD34+ hematopoietic stem and progenitor cells (HSPCs) led to very efficient and persistent gene deletion in vivo.
Tian-Li Xiao, J. R. Brewer, Maximillian J. Carlino et al.
· Journal of Clinical Investig... · 0 citations