CRISPR/Cas9, a promising approach for the treatment of β-thalassemia: a systematic review

The CRISPR/Cas9 technique is easily programmable, fast, more powerful, and efficient at generating a mutation compared to previous gene therapy methods. beta-thalassemia is the most common autosomal recessive disorder worldwide. Appropriate genomic changes in the beta gene can be modified to alleviate the symptoms of the disease using the CRISPR/Cas9 system. PubMed/Medline, Scopus, Web of Science, and SID databases were searched in Persian and English from February 2000 to September 2022. Finally, 39 articles had inclusion criteria which were reviewed by two separate individuals. Among the reviewed articles, articles were divided into three categories. In the first group, studies attemped to increase the expression of gamma-globin and production of hemoglobin F. The strategy of second group of studies were the reduction of the alpha-globin chain to prevent hemolysis of RBCs by accumulation of excessive alpha-globins. The third group corrected the mutations causing beta-thalassemia. Studies have shown that the genome of beta-thalassemia patients can be modified using the CRISPR/Cas9 technique, and this approach might be promising for the treatment of beta-thalassemia.