King Faisal Specialist Hospital and Research Centre in Saudi Arabia has successfully applied advanced gene therapy for eight patients with hereditary bleeding disorder (hemophilia), demonstrating its effectiveness through a single dose that increased their missing clotting factor to levels that allow them to engage in daily life without the need for preventive treatment. This marks a revolutionary change in the quality of life for the patients and their families.
The gene therapy for hemophilia involves injecting patients with new working genes that enable their bodies to produce the deficient clotting factor on their own. The new genes provide instructions for the patients' bodies on how to create the blood clotting factor after they receive a single intravenous treatment.
This condition arises from a genetic defect that prevents the affected individual's body from producing a specific clotting factor, typically requiring them to receive regular injections of clotting factors intravenously every three to five days for life to manage bleeding episodes. Hemophilia is caused by a mutation or change in one of the genes that provides instructions for producing the blood protein (clotting factor) necessary for forming a blood clot to stop bleeding. The genetic mutation leads to the absence of this vital clotting factor.
The disease results in severe bleeding in various parts of the body, such as the joints, which can cause swelling, pain, and joint discomfort, or it may lead to muscle bleeding or internal bleeding in the head.
This achievement is part of the hospital's ongoing efforts to harness every available resource and bring in the latest technologies to enhance outcomes, patient experiences, and operational efficiency, making it the preferred choice for patients seeking specialized healthcare. King Faisal Specialist Hospital and Research Centre is one of the world's leading institutions in providing specialized healthcare, pioneering innovation, and serving as an advanced center for research and medical education. It aims to develop medical technologies and elevate healthcare standards globally in partnership with major local, regional, and international institutions to achieve world-class services in clinical, research, and educational fields.
