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Article

Trehalose Treatment in Zebrafish Model of Lafora Disease.

International journal of molecular sciences - 20 Jun 2022

Della Vecchia Stefania, Ogi Asahi, Licitra Rosario, Abramo Francesca, Nardi Gabriele, Mero Serena, Landi Silvia, Battini Roberta, Sicca Federico, Ratto Gian Michele, Santorelli Filippo Maria, Marchese Maria

Abstract excerpt

Mutations in the EPM2A gene encoding laforin cause Lafora disease (LD), a progressive myoclonic epilepsy characterized by drug-resistant seizures and progressive neurological impairment. To date, rodents are the only available models for studying LD; however, their use for drug screening is limited by regulatory restrictions and high breeding costs. To investigate the role of laforin loss of function in early...

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Identifiers

PMID: 35743315
DOI: 10.3390/ijms23126874
PMCID: PMC9224929

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