UAE Launches Gene Therapy Projects to Treat Autism and Rare Brain Disorders in Children
The United Arab Emirates (UAE) has introduced two groundbreaking gene therapy programs aimed at treating autism and rare neurogenetic brain disorders in children. Led by Emirates Health Services, these initiatives represent a major shift from managing symptoms to targeting the genetic causes of disease.
This move places the UAE among the leading countries advancing paediatric neurological care and precision medicine in the region.
Focus on Genetic Causes of Autism
One of the initiatives targets genetic mutations linked to autism and epilepsy. The project is being developed in partnership with the Children’s Hospital of Philadelphia, a globally respected paediatric research institution.
Researchers are working to create improved gene delivery systems that can accurately target specific brain cells. This approach aims to:
Increase treatment effectiveness
Reduce medication dosage
Lower long-term healthcare costs
The research also includes Beta-Propeller Protein-Associated Neurodegeneration (BPAN) — a rare neurological disorder that begins with developmental delays and seizures and can later lead to severe cognitive and motor decline.
By addressing the disease at the genetic level, scientists hope to change its progression rather than simply manage symptoms.
Research Advancements for Neurodegenerative Diseases
The second program focuses on rare neurodegenerative disorders. Researchers will explore:
Gene repair techniques
Cell-based disease models
Biomarkers to track disease progression and treatment response
Artificial intelligence (AI) will be used to analyse genomic data, helping doctors improve diagnosis and identify high-risk children at an earlier stage.
Data from the Emirati Genome Programme will play a vital role in enabling personalized treatments. The integration of genomics and AI reflects a broader healthcare strategy focused on predictive and preventive medicine.
Key Facts for Competitive Exams
Gene therapy involves modifying or replacing faulty genes to treat disease.
BPAN is a rare inherited neurological disorder.
The Emirati Genome Programme maps genetic variations within the UAE population.
Precision medicine customizes treatment based on an individual’s genetic profile.
A Strategic Shift Toward Innovation in Healthcare
Health officials describe these initiatives as part of a long-term transformation toward innovation-driven healthcare. By investing in biotechnology, global partnerships, and data analytics, the UAE aims to strengthen domestic medical expertise while improving the quality of life for affected families.
These projects reflect a global shift toward treating inherited neurological disorders through targeted genetic therapies rather than conventional symptom-based care.
