Applied Stem Cell Research in Sickle Cell Disease Publisher



Kiumarsi A¹ ; Rostami T² ; Mohammadi MS³
Source: Comprehensive Hematology and Stem Cell Research: Volume 1-5 Published:2024

Abstract

Sickle cell disease (SCD) is one of the most common recessive genetic disease, leading to significant morbidity and mortality, worldwide. Even though chronic red blood cells transfusion and drugs such as hydroxyurea ameliorate symptoms of SCD, development of a curative treatment for this monogenic disease is very desirable. Hematopoietic stem cell transplantation from an allogeneic donor has been successfully employed for treating SCD but its application is limited because of a lack of immunologically matched donors and complications such as graft versus host disease. Therapeutic strategies that are capable of correcting the causative mutation permanently in stem cells which could expand and differentiate into erythrocytes with the ability of producing the corrected globin are being explored. Moreover, by genome editing methods, correction of a genetic mutation in the native genome without alterations in other parts of the human genome is possible. However, these promising techniques still have safety and efficacy issues that must be addressed before being translated into clinical practice. This review concentrates on the cell and gene therapies which are being currently addressed for patients with SCD and the novel strategies aiming to improve the safety and efficiency of the potentially curative treatments. © 2024 Elsevier Inc. All rights are reserved.