UAE Patient With Heart Failure, Thalassemia Treated With Gene Therapy After Transplant

(MENAFN- Khaleej Times) Beta-thalassemia is more common in the Middle East than in many other parts of the world, and health authorities have long highlighted it as a significant inherited blood disorder

• PUBLISHED: Wed 18 Feb 2026, 12:19 PM

• By:
• Haneen Dajani

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A patient from the UAE with beta-thalassemia and severe heart failure has successfully undergone gene therapy after first receiving a heart transplant - a sequence of treatment that many centres would consider too complex or high-risk to attempt.

According to Rabi Hanna, paediatric cellular therapy specialist at Cleveland Clinic, the patient was treated using their own stem cells, allowing doctors to address both the underlying genetic disease and the cardiac condition without the need for long-term immunosuppression following gene therapy.

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“In many centres, this level of complexity would exclude a patient from gene therapy altogether,” Hanna said.“But because the treatment relies on the patient's own stem cells, we were able to successfully treat both the heart failure and the genetic disease. Today, the patient is doing very well.”

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What gene therapy actually involves

Gene therapy aims to correct a faulty gene that causes disease. In conditions such as beta-thalassemia and sickle cell disease, this faulty gene affects the body's ability to produce healthy red blood cells.

One of the most widely used approaches today is CRISPR, a gene-editing technology that works like molecular scissors, allowing doctors to precisely alter DNA inside a patient's stem cells. These corrected stem cells are then returned to the body, where they can produce healthy blood cells.

However, Hanna stressed that gene therapy is not a single treatment, but a long and carefully managed process that can take nine to 15 months from start to finish.

Demanding but carefully controlled process

The journey begins with stem cell collection. Some patients require only one collection cycle, while others may need several rounds over months.

Once collected, the stem cells are sent for gene editing and extensive safety testing - a manufacturing phase that alone can take close to six months.

Before the modified cells can be infused back into the patient, doctors must administer myeloablative chemotherapy- a strong form of chemotherapy designed to clear out the bone marrow and make space for the corrected cells to grow. While effective, this step can cause side effects such as mouth sores, infection risk and longer-term concerns including fertility issues, all of which require careful counselling and monitoring.

“This is why preparation and realistic expectations are so important,” Hanna said.“Patients and families need to understand both the short-term challenges and the lifelong follow-up involved.”

Why this matters in the region

Beta-thalassemia is more common in the Middle East than in many other parts of the world, and regional health authorities have long highlighted the condition as a significant inherited blood disorder affecting families across the Gulf and wider region.

Advances that safely expand access to curative treatments therefore carry particular relevance for patients from the UAE and neighbouring countries.

New gene-editing techniques show faster recovery

Alongside traditional CRISPR approaches, doctors are increasingly using base editing, a newer form of gene editing that changes a single 'letter' in DNA without cutting both strands of the genetic code. This added precision appears to keep stem cells healthier.

Data presented at American Society of Hematology meetings from recent BEAM studies suggest base editing leads to faster recovery, fewer complications and shorter hospital stays.

In one recent case treated using base editing, Hanna said the patient's corrected cells successfully took hold by day 14, compared to the more typical timeline of around day 27. The quicker recovery allowed the patient to leave hospital sooner and return to daily life well before day 100.

Moving towards chemotherapy-free gene therapy

Researchers are now working toward an even more significant shift: in vivo gene editing, where genetic changes are made directly inside the body, potentially eliminating the need for chemotherapy altogether.

Another promising approach involves antibody-based conditioning, which could replace chemotherapy with a far less toxic method of preparing the body for gene therapy.

“These innovations represent the next evolution of gene therapy,” Hanna said.“The aim is to make treatment safer, less burdensome and accessible to more patients.”

Treating the patient, not just the disease

Hanna emphasised that long-term success depends on integrated care, involving not just specialist centres but also local hematologists, mental health professionals and primary care teams.

Patients require structured follow-up at one year after treatment and annually thereafter to monitor for late effects - much of which can be managed close to home with specialist support.

“Gene therapy is not just about fixing a gene,” Hanna said.“It's about restoring quality of life, and that requires a coordinated, long-term approach.”

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