Stem Cell-Derived Therapy for Sickle Cell Anemia

Abstract

Background:The genetic blood disease known as sickle cell anemia (SCA) results from a mutation in the HBB gene, which codes for the β-globin component of hemoglobin. Aberrant hemoglobin S (HbS) produced by this mutation causes red blood cell abnormalities, a shortened lifetime, and problems with vaso-occlusive pathways. Hydroxyurea and bone marrow transplants are two current therapies with limited availability of donors and efficacy. Targeting the fundamental genetic abnormality, induced pluripotent stem cells (iPSCs) and hematopoietic stem cells (HSCs) both show potential as a therapeutic approach.
Aim:The project aims to develop and maximize protocols for genetically modifying HSCs or iPSCs to fix the HBB gene mutation, evaluate the differentiation potential of these cells into functional erythroid cells, and test their therapeutic efficacy in vitro and preclinical models, so assessing the efficacy of stem cell-derived therapies for sickle cell anemia.
Conclusion:Stem cell-derived treatments provide a revolutionary solution for sickle cell anemia. Reintroduced into patients, genetically engineered HSCs or iPSCs can provide a sustainable source of healthy red blood cells, hence perhaps curing the disease. From initial studies, successful gene repair and differentiation into functional erythroid cells seem to follow. Still unsolved, meanwhile, are questions on improving gene-editing efficiency, ensuring transplanting safety, and managing immunological compatibility. With more research, stem cell treatments may significantly improve the outcomes of sickle cell anemia patients.