Gene replacement therapy for Lafora disease in the Epm2a-/- mouse model
Luis Zafra‐Puerta, Daniel F. Burgos, Nerea Iglesias‐Cabeza, Juan González‐Fernández, Gema Sánchez‐Martín, Marina P. Sánchez, José María Serratosa
This study uses gene replacement therapy in Epm2a-/- mice to treat Lafora disease, showing improvements in neurological symptoms and reduced Lafora body formation.
bioRxiv · Dec 14, 2023 · PreprintNeuroscience