IJPMBS 2026 Vol.15(2): 29-33
doi: 10.18178/ijpmbs.15.2.29-33
doi: 10.18178/ijpmbs.15.2.29-33
Research Progress and Future Directions of CRISPR Gene Editing Technology in β-thalassemia
Keqi Zhou
Xihua Univerisity, Chengdu, China
Email: a2265477625@163.com
Email: a2265477625@163.com
Manuscript received May 27, 2026; accepted June 2, 2026; published June 9, 2026.
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.
Keywords—β-thalassemia, CRISPR gene editing technology, HBB, BCL11A
Cite: Wen Li, "Research Progress and Future Directions of CRISPR Gene Editing Technology in β-thalassemia," International Journal of Pharma Medicine and Biological Sciences, Vol. 15, No. 2, pp. 29-33, 2026.
Copyright © 2026 by the authors. This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).
Cite: Wen Li, "Research Progress and Future Directions of CRISPR Gene Editing Technology in β-thalassemia," International Journal of Pharma Medicine and Biological Sciences, Vol. 15, No. 2, pp. 29-33, 2026.
Copyright © 2026 by the authors. This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).
