Groundbreaking Gene Therapy Saves Children’s Vision

Cleveland Clinic Abu Dhabi is the First and Only Hospital in the UAE to Offer Innovative Treatment

​Two Emirati children are among the first patients in the world to benefit from a new genetic therapy to treat a rare, formerly untreatable condition that leads to blindness. 

The two sisters, Alia aged 13 and Hessa, aged 11, received the vision-saving treatment at Cleveland Clinic Abu Dhabi’s Eye Institute, one of a select number of facilities equipped to administer it. 

With the support of the Department of Health, the sisters became the first of several patients in the country eligible to receive the treatment.  

The sisters suffered from RPE65-related retinal dystrophy, a genetic disorder in which the RPE65 protein is lacking. As this protein is required to maintain the retina, left untreated the disease leads to progressive damage to eyesight and permanent blindness. 

Using a bioengineered non-pathogenic virus, the groundbreaking genetic therapy delivers normal copies of the RPE65 gene to the eye, allowing for the production of the protein, helping to preserve and even repair the tissue of the retina. 

“The prospect of slowly losing one’s vision from an untreatable condition is traumatic for both children and their parents. This genetic therapy means we can now replace the faulty gene in the eye, saving and even improving the vision of an individual who would otherwise have eventual irreversible blindness. This is extremely significant,” says Dr. Arif Khan, a pediatric ophthalmologist and ocular geneticist at Cleveland Clinic Abu Dhabi. Dr. Khan diagnosed the disease in the two sisters and led the Cleveland Clinic Abu Dhabi initiative to bring the treatment to Abu Dhabi. 

RPE65- related retinal dystrophy occurs when a child inherits two copies of the defective RPE65 gene from both parents. Each asymptomatic parent carries one defective copy of the gene. While rare, it is more common in the Gulf due to unique factors in the population. The deficiency is suspected by careful clinical examination and is confirmed by specialized genetic testing. 

Genetic therapy to treat the condition is best performed at a young age, before significant retinal damage has occurred. The therapy was only recently approved by the Ministry of Health & Prevention in Abu Dhabi following extensive clinical trials in the United States.  Since undergoing their treatment, Alia and Hessa have shown improvement and continue to be closely monitored by their care team at Cleveland Clinic Abu Dhabi.

“I am so proud that my children have been able to benefit from this treatment here in the UAE. The idea of them losing their vision terrified me and now they can continue to focus on their schooling to give back to this great nation that has given us so much. I am so thankful to the doctors at Cleveland Clinic Abu Dhabi and the government for helping my family,” says Fatima, Alia and Hessa’s mother. 

Cleveland Clinic Abu Dhabi worked with the Department of Health, the medication’s distributor and a number of insurance partners to make the treatment available. 

For more information about Cleveland Clinic Abu Dhabi’s Eye Institute or to schedule an appointment, visit www.clevelandclinicabudhabi.ae​ or call 800 CCAD (2223).