Description
Our research activity focuses on studying mechanisms of pathogenesis and
discovering potential drug therapies for diseases of genetic origin, in particular Myotonic Dystrophy (DM1 and DM2), Spinal Muscular Atrophy (SMA) and Limb-Girdle Muscular Dystrophy (LGMDD2).
Keywords
Muscleblind, Drosophila, miRNA, biomarker, Myotonic Dystrophy, Spinal Muscular Atrophy, alternative splicing, toxic RNA, drug discovery; therapy development, antisense oligonucleotides, drug repurposing, RNA drugs, SMN, TNPO3, LGMDD2, muscular atrophy, MSI2, cachexia.
Frascati classification
