Genome editing in therapy: immunological context

Genome editing of induced pluripotent stem cells (iPSc) or hematopoietic stem cells (HSC) from the patient’s bone marrow is proposed as part of the gene therapy. It seems that this approach allows obtaining autogenous cells. However, it will not always be an autologous transplant in the strict sense of this term. Transplant rejection will be more likely to occur in recessive diseases caused by nonsense mutations, since in these patients’ organism the protein that appears after genome editing will be “foreign” to the host, although it will be produced by autogenous cells. Apart from the aspect of the quite unique transplant situation described above, the use of iPSc derivatives or HSC cells with an edited genome seems to be more promising in diseases in which symptoms depend on blood cell dysfunction than, for example, in muscular dystrophy or cystic fibrosis. Genome editing is also used to refine autogenous T cells with chimeric antigen receptor T-cells (CAR-T). Finally, genome editing in combination with iPSc technology should enable the production of the so-called universal cells for transplantation, including universal CAR-T. Genome editing is therefore particularly important in an immunological context.