UAE launches gene therapy projects for autism and rare brain disorders in children

The United Arab Emirates has unveiled two groundbreaking gene therapy projects focused on treating serious neurological and genetic conditions in children. These efforts mark a major shift from conventional symptom-based care to tackling the underlying genetic causes of disease, potentially transforming neurodevelopmental healthcare in the region.

One key project focuses on genetic causes linked to autism and epilepsy. It is being developed in collaboration with the Children’s Hospital of Philadelphia, a paediatric research centre. Scientists will work on enhanced gene delivery systems that target specific brain cells, which could improve treatment effectiveness, reduce required doses, increase overall safety, and lower long-term treatment costs.

The project specifically includes rare conditions such as Beta-Propeller Protein-Associated Neurodegeneration (BPAN), a disorder that often begins in childhood with autism-like symptoms and seizures before progressing to serious motor and cognitive decline. This strategy represents a major scientific shift because it aims to treat the root genetic causes rather than just managing symptoms.

The second gene therapy initiative centres on neurodegenerative diseases. Researchers will explore methods to repair faulty genes and use advanced techniques such as cell-based therapy models to study disease progression, biomarker identification to track disease and treatment response, and tools to better understand how brain diseases evolve over time.

Artificial intelligence and data analytics will help researchers make sense of genetic information collected through national programmes like the Emirati Genome Programme, enabling earlier diagnosis and intervention for children at higher risk. The approach relies on cutting-edge technology and global expertise to refine treatment precision and open doors for future inherited disease therapies.

Leading experts at Emirates Health Services described the projects as part of a strategic shift in healthcare, moving from traditional care models to evidence-based, innovation-driven treatments that directly address the causes of genetic disease. The direction aims to improve quality of life and local capabilities in advanced medicine and biotechnology, while integrating scientific research with responsible clinical application to reduce the psychological, social, and economic burdens families face.