NeuroscienceJournal of Translational MedicinePeer-reviewed
Reversible synaptic deficits in early-stage Batten disease
Masood Ahmad Wani, Chloe M. Hall, Thomas Mittmann…·Jakob von Engelhardt
This study shows that CLN3 is essential for synaptic function in Batten disease and that early gene therapy can reverse synaptic deficits.
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