video: Targeted gene therapy is thought to be the way ahead for treating drug-resistant epilepsy, a condition affecting about one-third of patients with epilepsy, despite medication. Now, researchers led by Professor Hirokazu Hirai from Gunma University, Japan, have developed cmGAD67 – a compact promoter that selectively targets inhibitory neurons in the brain to restore the excitatory–inhibitory balance, essential for the brain to function normally. It offers a promising avenue as a compact and efficient platform for targeted gene therapy. Reference Title of original paper: A compact GAD67 promoter enables inhibitory neuron-targeted AAV gene therapy for seizure suppression Journal: Molecular Therapy DOI: https://doi.org/10.1016/j.ymthe.2026.06.007
Credit: Professor Hirokazu Hirai from Gunma University, Japan
Journal
Molecular Therapy