Research Progress and Future Directions of CRISPR Gene Editing Technology in β-thalassemia

Abstract

β-thalassemia is a hereditary hemolytic illness where the synthesis of hemoglobin gets reduced. It mainly comes about because of mutations in the β-globin gene. In the recent few years, CRISPR gene editing technology has been brought in and used to correct the mutations related to β-thalassemia. This paper gives a full look at how CRISPR gene editing technology has been applied in treating β-thalassemia over the past five years. The mechanisms and the latest clinical progress of CRISPR gene editing technology when it comes to treating this disease are explored in a systematic manner through three ways: mending the HBB mutation sites, aiming at the BCL11A enhancer to get fetal hemoglobin activated, and holding back the expression of α-globulin. On November 16, 2023, the UK Medicines and Healthcare Products Regulatory Agency gave the green light to the therapeutic drug Casgevy, which makes use of CRISPR-Cas9 gene editing technology, for treating transfusion-dependent β-thalassemia patients. This marks the start of a new era in gene therapy. But CRISPR gene editing technology still has risks like off-target effects. The appearance of new technologies such as base editors brings a new angle for treatment.