Genetically modified hematopoietic stem cells for treatment of hematological diseases
Bulgarian Medical Journal, 2025, 19(3), 27-32.
Т. Vekov1, B. Spasov1, L. Dimova1, V. Belcheva2
1 MHAT „Heart and Brain“ – Pleven
2 Trakia University – Stara Zagora
Abstract. The use of allogenic hematopoietic stem cells (HSCs) for treatment of genetic hematological diseases became a clinical standard; however, it is restricted by the presence of eligible donors and potential immunological complications. The majority of inherited hematological diseases such as primary immunodeficiency, hemoglobinopathies, metabolic disorders, congenital cytopenias and defects of stem cells, can be treated by transplantation of allogenic HSCs. Transplanted, genetically normal HSCs can serve as a constant source of all lines of blood cells, eliminating these diseases with a single treatment (transplantation) and lifelong benefits. At the same time, although generally allogenic transplantation of HSCs is with a high proportion of success, the results are less successful with non-identical donors. There is a constant progress with the methods for reduction of the response of a graft to a recipient in allogenic transplantation of HSCs, including improved modification of the graft by the removal of selective Т-cell populations (TCR α/β depletion) and the use of post-transplantation cyclophosphamide.
Key words: genetically modified hematopoietic stem cells, hematological diseases
Address for correspondence: Prof. Toni Vekov, e-mail: t.vekov.mu.pleven@abv.bg