UAE Launches Gene Therapy Projects for Autism Treatment

Precision Medicine Push Targets Rare Brain Disorders in UAE

The United Arab Emirates has introduced two advanced gene therapy initiatives designed to address autism and rare neurogenetic brain disorders in children. Spearheaded by Emirates Health Services , the programmes reflect a strategic transition from conventional symptom management to precision medicine focused on genetic causes.

Targeting Genetic Drivers of Autism

One initiative centres on genetic mutations associated with autism and epilepsy. The project is being developed in collaboration with the Children’s Hospital of Philadelphia , recognised globally for paediatric research. Researchers are working on improved gene delivery technologies capable of precisely targeting specific brain cells.

This approach aims to enhance therapeutic effectiveness, minimise dosage requirements, and optimise long-term treatment costs. Among the disorders under study is Beta-Propeller Protein-Associated Neurodegeneration (BPAN), a rare inherited neurological condition characterised by developmental delays and seizures, often progressing to severe cognitive and motor impairment.

Research on Rare Neurodegenerative Diseases

The second initiative focuses on rare neurodegenerative disorders. Scientists will explore gene repair mechanisms, develop cell-based disease models, and identify biomarkers to track disease progression and treatment outcomes. Artificial intelligence tools will support genomic data analysis, facilitating earlier diagnosis and risk prediction.

Data integration from the Emirati Genome Programme will enable personalised medical interventions. The combination of genomics and AI aligns with broader healthcare objectives centred on predictive and preventive medicine.

Strategic Healthcare Advancement

Officials describe these projects as part of a long-term innovation-driven healthcare transformation. Investments in biotechnology, global partnerships, and advanced analytics aim to build domestic expertise while improving patient outcomes.

Exam-Focused Key Points

• Gene therapy modifies or replaces defective genes.

• Precision medicine customises treatment using genetic data.

• BPAN is a rare inherited neurodegenerative disorder.

• AI increasingly supports genomic diagnostics.

• National genome projects aid personalised healthcare.