Progress of CRISPR-Cas9 in Treatment of β-thalassemia

Abstract

β-thalassemia is a common genetic blood disorder, including three clinical and hemtological conditions: the thalassemia intermedia, β-thalassemia carrier state, and thalassemia major. It is widely distributed in the tropical, subtropical and mediterranean regions, and It has grown to be a significant global public health issue. Currently, the sole curative treatment for β-thalassemia is hematopoietic stem cell transplantation (HSCT), but the development of the HSCT is limited by the lack of an immunomatched donor and the high cost. Considering the advancement of gene editing technology in recent years, it is possible to cure the β-thalassemia with low cost and higher chances of surviving. CRISPR-Cas9 system, since its characteristics of simple operation, targeting specificity and low price, has been widely used in the field of life science and medical science. Currently, the study of CRISPR-Cas9 technology in the treatment of β-thalassemia has made a partial progress. This review systematically introduces the application of CRISPR-Cas9 technology in the treatment of β-thalassemia.