Article

A compact glutamic acid decarboxylase 67 promoter enables inhibitory neuron-targeted AAV gene therapy for treatment-resistant epilepsy

2025-12-10

Abstract excerpt

<h4>ABSTRACT</h4> Epilepsy often becomes treatment-resistant, partly due to impaired inhibitory neurotransmission and reduced γ-aminobutyric acid (GABA) function. Enhancing inhibitory neuron activity via gene therapy may restore excitation–inhibition (E/I) balance. We developed a compact 410-bp glutamic acid decarboxylase 67 promoter (cmGAD67) that enables strong, selective transgene expression in inhibitory neur...

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Literature Corpus work: e428107f-8454-5b4b-b7b1-33764e85ee49
DOI: 10.64898/2025.12.07.692482

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