Matos, Liliana2018-03-292018-03-292017-03-22http://hdl.handle.net/10400.18/5488Short term scientific mission about therapeutic strategies to modulate splicing in lysosomal storage disorders.engU1snRNA Splicing TherapiesLentiviral TransductionLysosomal Storage DisordersDoenças GenéticasDevelopment of antisense U1 snRNA-mediated therapeutic strategies to modulate splicing in lysosomal storage disorderslecture