Riyadh: Saudi Arabia’s King Abdulaziz Medical City (KAMC) in Riyadh has made significant strides in treating thalassemia using CRISPR gene editing technology. In a groundbreaking move, KAMC successfully treated a 13-year-old patient with thalassemia major using Casgevy gene therapy, marking the first successful application of this advanced therapy outside clinical trials.
The patient, who had relied on blood transfusions every three weeks since birth, has now fully recovered following the gene cell transplantation. This achievement represents a major advancement in the treatment of genetic disorders, offering a potential permanent solution to thalassemia major, a severe blood disorder caused by genetic mutations.
The successful use of CRISPR technology not only eliminates the patient’s dependency on regular transfusions but also demonstrates the transformative potential of gene therapy for treating genetic diseases. The Ministry of National Guard Health Affairs (MNGHA) is now preparing to extend these innovative treatments to more patients suffering from sickle cell anemia and thalassemia.
This breakthrough highlights Saudi Arabia’s commitment to advancing medical science and positions the country as a global leader in gene therapy. The successful integration of CRISPR technology into clinical practice sets a new standard for global healthcare innovation and offers hope for many individuals affected by genetic disorders.
