A Comprehensive Review on Stem Cell Therapy and CRISPR/Cas Technology in the Treatment of Thalassemia
TL;DR
A variety of research is synthesized, examining the therapeutic potential of stem cell therapy and CRISPR/Cas technology in the treatment of thalassemia, highlighting the use of stem cell therapy and CRISPR/Cas gene editing technology as alternative treatment options.
Abstract
Thalassemia is a widespread hereditary blood disorder, characterized by defective or impaired production of the ???? or ????-globin chains that make up hemoglobin. This imbalance leads to ineffective erythropoiesis, anemia and additional associated symptoms. Current therapeutic strategies largely rely on hematopoietic stem cell (HSC) transplantation as a curative approach. However, recent research illustrates the use of stem cell therapy and CRISPR/Cas gene editing technology as alternative treatment options. Beyond HSCs, mesenchymal stem cells (MSCs) can potentially ameliorate clinical manifestations due to their ability to differentiate into diverse cell types, while induced pluripotent stem cells (iPSCs) are re-engineered somatic cells that can be genetically modified for thalassemia treatment. Advances in clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) include the reactivation of fetal hemoglobin through editing of the erythroid-specific enhancer of BCL11A, direct correction of HBB mutations, and genetic alterations in iPSCs. This review synthesizes a variety of research, examining the therapeutic potential of stem cell therapy and CRISPR/Cas technology in the treatment of thalassemia.