Figure 1.

Overview of exemplary hereditary disorders potentially curable by editing of HSCs. Genes associated with each disease are indicated in parentheses. The cell types given indicate where protein expression or phenotypic correction are most apparent. Note that combined immunodeficiencies affect B cell function even when presenting with a B⁺ phenotype. Monocytes and macrophages (MΦ) may also act on cells and for disease correction outside the hematopoietic system (not shown). RBC—red blood cell, NK cell—natural killer cell.