Development of an antisense-mediated exon skipping approach as a therapeutic option for the ML II-causing mutation c.3503

Matos, LilianaVilela, ReginaCoutinho, Maria FranciscaGaspar, PauloAlves, Sandra2018-03-292018-03-292017-09http://hdl.handle.net/10400.18/5501Lysosomal storage disorders (LSDs) are a group of a rare inherited metabolic disorders.engMucolipidose Tipo IITerapias de SplicingDoenças GenéticasDevelopment of an antisense-mediated exon skipping approach as a therapeutic option for the ML II-causing mutation c.3503_3504delTCconference object