ArticleJournal of translational medicine2026
Reversible synaptic deficits in early-stage batten disease.
Article in Journal of translational medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundJuvenile neuronal ceroid lipofuscinosis (JNCL, Batten Disease) is a childhood-onset, neurodegenerative, lysosomal storage disorder caused by mutations in the lysosomal gene CLN3. Progressive cognitive decline is characteristic clinical feature, and no definitive treatment is currently available. The neuronal function of CLN3 is unknown, and the pathomechanisms leading to cognitive impairment are poorly understood hindering the development of targeted therapies.
methodsWhole-cell patch clamp and high-density microelectrode array recordings were performed in acute brain slices from Cln3
resultsLoss of CLN3 caused defective synaptic vesicle release and reduced synaptic strength, reflecting impairments in both pre- and postsynaptic function in Cln3
conclusionsOur findings demonstrate a critical requirement for CLN3 in maintaining synaptic function and show that targeted gene therapy can restore established functional deficits in Cln3-deficient mice. This suggests that the therapeutic window may extend to stages already characterized by functional impairments, raising hope that targeted interventions could not only slow disease progress but to also potentially restore neuronal function and thereby improve clinical outcome. Moreover, these early synaptic deficits provide sensitive and robust functional readouts that can support preclinical research.
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