KFSH&RC applies advanced gene therapy to patients with hemophilia

The King Faisal Specialist Hospital and Research Centre (KFSH&RC) has "successfully administered an advanced gene therapy" to eight patients suffering from hemophilia, a genetic bleeding disorder, according to a press release issued by the center, SPA reads.

The hospital has demonstrated the "therapy's efficacy in substantially elevating the deficient clotting factor with just a single dose. This increase enables patients to engage in daily activities without the constant requirement for preventive treatment", the release said, adding that this is going to improve the quality of life of patients with hemophilia.

Gene therapy for hemophilia works by introducing new, functional genes into the patient's body, enabling it to independently manufacture the previously deficient clotting factor. The new genes instruct the patient's own cells how to synthesize the blood-clotting factor in a process that is typically achieved with just one therapeutic dose delivered intravenously.

Hemophilia arises from a genetic mutation that affects the genes responsible for instructing the production of a specific blood protein: the clotting factor. Patients with this condition typically require lifelong management, which involves regular injections of the clotting factor directly into the bloodstream every three to five days to prevent and control bleeding episodes, according to the press release.

This disorder causes significant bleeding in various body parts, particularly the joints, which can lead to symptoms like swelling, pain, and limited joint mobility. It can also cause bleeding in the muscles and potentially dangerous internal bleeding, in the brain.

The KFSH&RC's achievement is testament to its commitment to leveraging every available resource and integrating cutting-edge technologies to improve treatment outcomes, patient experience, and the efficiency of healthcare delivery.

KFSH&RC is globally recognized for its "exceptional specialized healthcare services and groundbreaking innovations". This makes it an advanced hub for medical research and education, committed, according to the release, to advancing medical technologies and raising the standard of healthcare on a global scale.