Pioneering gene therapy programs aim to treat neurological disorders in the UAE.
Dubai: The UAE is stepping up its advancements in medical innovation, unveiling two groundbreaking gene therapy projects designed to treat rare neurological and genetic disorders in children, moving healthcare focus from symptom management to addressing the root causes of disease.
The Emirates Health Services (EHS) stated that the initiatives focus on monogenic autism and innovative genetic therapies for neurodegenerative disorders, supporting the UAE’s strategy to expand investment in advanced medical technologies and enhance long-term healthcare outcomes.
The first project, currently under development, targets genetic causes associated with autism and epilepsy in children. It addresses rare and severe conditions such as Beta-Propeller Protein-Associated Neurodegeneration (BPAN), a disorder that typically manifests early in life with autism spectrum symptoms and seizures, and later progresses to motor and cognitive decline during adolescence.
The EHS stated that the new therapeutic approach will employ advanced gene delivery systems designed to target specific brain cells, potentially improving treatment effectiveness, reducing required dosages, and enhancing safety. The strategy is also expected to lower long-term treatment costs and pave the way for addressing other 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 pediatric research centers. This partnership seeks to combine global expertise with local research capabilities to expand treatment options for children in the UAE affected by rare neurological conditions.
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 disease progression. Researchers will also identify biological markers in the brain to monitor disease development and measure treatment responses more accurately.
Artificial intelligence and data-driven tools will play a central role in the program. By analysing data from the Emirati Genome Programme, specialists aim to identify children at higher risk of genetic disorders, enabling earlier diagnosis and timely intervention.
Dr Issam Al Zarooni, Executive Director of Medical Services at EHS, said the projects mark a strategic shift toward innovation-driven healthcare. “Investing in gene therapy reflects a move from traditional care to advanced treatment models that directly target the genetic causes of disease. This approach aligns with national health strategies to enhance quality of life and develop local expertise in advanced medicine and biotechnology,” he said.
Dr Noor Al Mheiri, Director of Mental Health and Visiting Physicians at EHS, added that the program integrates scientific research with responsible clinical application. She noted that the goal is to improve early diagnosis, enhance treatment response, and reduce the psychological and social burden on families through evidence-based, biomarker-driven therapies.
