Gene therapy in Abu Dhabi frees patient from transfusions — The National

In Abu Dhabi, UAE, a 43-year-old patient with transfusion-dependent thalassaemia no longer needs regular blood transfusions after receiving gene therapy treatment. The patient, identified only as Fahad, previously had to undergo the procedure every three to four weeks to maintain stable haemoglobin levels, The National reports.

Treatment using the patient’s own stem cells

About 18 months ago, Fahad was referred to Yas Clinic and the Abu Dhabi Stem Cells Center (ADSCC), where specialists were working to introduce gene therapy in the country. He was deemed a suitable candidate for this treatment.

Doctors collected the patient’s own stem cells, genetically modified them, and then reinfused them after preparatory therapy. Since then, the man has remained under medical supervision and is no longer dependent on regular blood transfusions.

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Course of the disease

Thalassaemia is a group of inherited blood disorders in which there is a deficiency of haemoglobin, the protein that carries oxygen. Low haemoglobin levels can cause anaemia, fatigue and shortness of breath. In Fahad’s case, the disease also led to iron overload and the need for continuous chelation therapy.

Yas Clinic’s chief medical officer, Maysoun Al Karam, said the case was an important milestone in expanding access to innovative treatments in the UAE. Yas Clinic haematologist Mohammed Abu Khaleika noted that gene therapy makes it possible to address the underlying cause of the disease using the patient’s own genetically modified cells, including for people who do not have a suitable donor for transplantation.