A compact glutamic acid decarboxylase 67 promoter enables inhibitory neuron-targeted AAV gene therapy for treatment-resistant epilepsy
Yuuki Fukai, Ayumu Konno, Nobutake Hosoi, Karin Miyakawa, Ryosuke Kaneko, Hirokazu Hirai
This paper develops a compact promoter for AAV gene therapy targeting inhibitory neurons, showing efficacy in treating epilepsy by restoring GABA function.
bioRxiv · Dec 10, 2025 · PreprintNeuroscience