TY  - JOUR
AU  - Zafra Puerta, Luis
AU  - Iglesias-Cabeza, Nerea
AU  - Burgos, Daniel F
AU  - Sciaccaluga, Miriam
AU  - González Fernández, Juan
AU  - Bellingacci, Laura
AU  - Canonichesi, Jacopo
AU  - Sánchez-Martín, Gema
AU  - Costa, Cinzia
AU  - Sánchez, Marina P
AU  - Serratosa, José M
PY  - 2024
DO  - 10.1016/j.ymthe.2024.05.032
UR  - https://hdl.handle.net/20.500.14352/113467
T2  - Molecular Therapy
AB  - Lafora disease is a rare and fatal form of progressive myoclonic epilepsy typically occurring early in adolescence. The disease results from mutations in the EPM2A gene, encoding laforin, or the EPM2B gene, encoding malin. Laforin and malin work...
LA  - eng
M2  - 2130
PB  - ELSEVIER
KW  - Epm2a(−/−) knockout mouse
KW  - gene therapy
KW  - laforin
KW  - polyglucosans
KW  - progressive myoclonic epilepsy
KW  - rAAV
TI  - Gene therapy for Lafora disease in the Epm2a-/- mouse model
TY  - journal article
VL  - 32
ER  -