Dapagliflozin ameliorates Lafora disease phenotype in a zebrafish model.

ABSTRACT: Lafora disease (LD) is an ultra-rare and still incurable neurodegenerative condition. Although several therapeutic strategies are being explored, including gene therapy, there are currently no treatments that can alleviate the course of the disease and slow its progression. Recently, gliflozins, a series of SGLT2 transporter inhibitors approved for use in type 2 diabetes mellitus, heart failure and chronic kidney disease, have been proposed as possible repositioning drugs for the treatment of LD. With this in mind, we tested dapagliflozin (50 µM), canagliflozin (2.5 µM) and empagliflozin (200 µM) in our epm2a^-/- zebrafish model, investigating their effects on pathological behaviour. In the case of dapagliflozin, we also investigated the possible mechanisms of action. Overall, th

SUBMITTER: Della Vecchia S 

PROVIDER: S-EPMC11794196 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature