Pioneering gene therapy projects target neurological disorders in UAE
Dubai: The UAE is accelerating its efforts in advanced medicine, unveiling two pioneering gene therapy projects aimed at treating rare neurological and genetic disorders in children and shifting care from managing symptoms to targeting the root causes of disease.
The Emirates Health Services (EHS) said the initiatives focus on monogenic autism and innovative genetic treatments for neurodegenerative conditions, supporting the country’s strategy to expand investment in advanced medical technologies and improve long-term healthcare outcomes.
The first project, currently under development, targets genetic causes linked to autism and epilepsy in children. It includes rare and severe conditions such as Beta-Propeller Protein-Associated Neurodegeneration (BPAN), a disorder that often presents early in life as autism spectrum symptoms and seizures before progressing to motor and cognitive decline during adolescence.
EHS said the new therapeutic approach will use enhanced gene delivery systems designed to target specific brain cells, which could improve effectiveness, reduce required dosage and increase safety. The strategy is also expected to lower long-term treatment costs and create pathways for tackling similar inherited disorders in the future.
The initiative is being developed in collaboration with the Children’s Hospital of Philadelphia, one of the world’s leading paediatric research centres. The partnership aims to combine global expertise with local research to expand treatment options for children affected by rare neurological conditions in the UAE.
A second project will focus on developing advanced genetic interventions for neurodegenerative diseases, including techniques to repair faulty genes, alongside the use of sophisticated disease models and cell-based therapies to better understand how such conditions progress. Researchers will also identify biological markers in the brain to track disease progression and measure response to treatment more accurately.
Artificial intelligence and data-driven tools will play a central role in the programme. By analysing data from the Emirati Genome Programme, specialists aim to identify children at higher risk of genetic disorders, enabling earlier diagnosis and intervention.
Dr Issam Al Zarooni, Executive Director of Medical Services at EHS, said the projects represent a strategic shift towards innovation-driven healthcare. “Investing in gene therapy reflects a move from traditional care to advanced treatment models that directly address the genetic causes of disease. This approach supports national health strategies to improve quality of life and build local capabilities in advanced medicine and biotechnology,” he said.
Dr Noor Al Mheiri, Director of Mental Health and Visiting Physicians at EHS, added that the programme integrates scientific research with responsible clinical application. She said the aim is to improve early diagnosis, enhance treatment response and reduce the psychological and social burden on families through evidence-based and biomarker-driven therapies.
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