UAE’s first gene therapy fixes eyesight of two Emirati sisters
January’s procedure improved vision of girls faced with irreversible blindness
Two Emirati sisters from Abu Dhabi have become the first recipients of gene therapy in the UAE. Alia, 13, and Hessa, 11, suffered from retinal damage due to a defective gene. The vision saving surgery was performed at Cleveland Clinic Abu Dhabi in January. “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,” said Dr Arif Khan, paediatric ophthalmologist and ocular geneticist.
Cleveland Clinic Abu Dhabi is only one of 10 centres worldwide, and the only one in the region, that is qualified to offer this procedure.
Alia and Hessa were born with RPE65-related retinal dystrophy, a rare disease known to affect one in 200,000 people worldwide.
Bioengineered virus used
Patients face gradual damage to their retinas and could eventually end up with irreversible blindness.
The gene therapy uses a vector — a bioengineered non-pathogenic virus — to deliver normal copies of the RPE65 gene to the eye.
My handwriting has definitely gotten better. I can’t tell what else will get better, but I already feel that this has been life-changing.”
Alia | One of the sisters who received gene therapy
“The procedure takes only about an hour, but it is very delicate. We use advanced microscopes to create openings in the white of the eye, then use a specialised canula to reach under the retina and inject the vector into the specific area, which can be as small as one-tenth of a millimeter,” explained Dr Emad Abboud, chief of the department of the posterior segment at the hospital’s Eye Institute.
Post-surgical recovery takes only about a week.
What the family said
“My handwriting has definitely gotten better,” Alia told Gulf News. “I can’t tell what else will get better but I already feel that this has been life-changing.”
The girls’ mother Fatima noticed the visual impairment when they were two months old. “The doctor pointed out that they were not focusing or making eye contact. So we were aware of the condition. It definitely made life very challenging, especially as their development was delayed,” she said.