DEFEAT-LD - Drug discovEry and repurposing to Find a trEAtmenT for Lafora Disease

Finanziato dall’Unione europea – Next Generation EU – PNRR M6C2 - Investimento 2.1 Valorizzazione e potenziamento della ricerca biomedica del SSN.

Assessing potential benefit of VAL-1221 on epilepsy and neurocognitive outcomes for patients with Lafora Disease (LD).
Find a potentially innovative treatment for LD by using cell and mouse models.
Provide an electrophysiological characterization of LD mouse models.
Activities
Evaluation of hyperexcitability and neurodegeneration mechanisms in LD models.
Investigation of LD models treated with VAL1221 and SINEUPs.
Compounds repurposing for potential clinical use in LD patient.
To investigate a SINEUP approach for the treatment of LD patients.
Results
Evaluation of VAL-1221 effects on LD patients.
Novel candidate compounds by high throughput screening.
Testing in cell cultures SINEUP non-coding RNAs to increase expression of alpha-amylase.
Neurophysiological effects of SINEUP, VAL-1221, and novel candidate compounds in LD animal models.

Importo di progetto Uniud 137.960,00 €
Importo di progetto totale 1.000.000,00 €.