Progress and development of CRISPR-Cas tool in the treatment of thalassemia

Abstract

-thalassemia is the worlds major hemoglobin disorders. It is a hereditary chronic hemolytic anemia caused by a break in the -globin peptide chain genes of hemoglobin, resulting in reduced or complete absence of synthesis of the -globin peptide chains. Patients with -thalassemia rely on blood transfusions for survival, which exacerbates the disease burden on national economies and the financial burden on patients in developing countries. Repeated blood transfusions can also lead to organ damage. The fetuss protein genes are usually silenced early and then replaced by adult protein genes. But mutations that cause gammaglobin to persist in the fetus can improve the electrocution of globin. Therefore, reactivating the protein genes of the fetus is the core of research. CRISPR/Cas9 is the common way to reduce the effects of disease by correcting disease-causing mutations, increasing or disrupting protein expression. CRISPR/Cas9 and considered effective new methods for generating hemoglobin. Hence, the main focus of this review is to explore the details of CRISPR/Cas9 in-thalassemia treatment in the context of Mediterranean -thalassemia.