"Breakthrough Gene Therapy Reverses SYNGAP1 Disorder Symptoms in Mice"

Researchers at the Allen Institute developed a groundbreaking gene therapy that reversed symptoms of SYNGAP1-related disorders in mice, marking a significant advance in treating neurological disorders.

• The Allen Institutes new gene therapy targets the SYNGAP1 gene, which is often compromised in individuals with SYNGAP1-related disorders, leading to severe symptoms like epilepsy and intellectual disability.
• Using an adeno-associated virus, the gene therapy successfully restored normal function in the SYNGAP1 gene in mouse models, demonstrating significant improvements in brain function and behavior.
• The study, published on October 9, 2025, highlights the potential of gene therapy to address debilitating neurological disorders, paving the way for future treatments in humans.