Towards a cure: using edited hematopoietic stem cells

Hematopoietic stem cell (HSC) transplantation has shown great promise in treating a vast spectrum of hereditary blood cell diseases and acquired disorders that affect hematopoietic and other tissues (1). However, allogeneic HSC transplantation (HSCT) is severely limited by the availability of matched donors and potential immunologic complications. Therefore, safe, efficient, and precise genome editing in autologous HSCs has become the “holy grail” of the field and stands as the paradigm of stem cell engineering.