Matos, LilianaCoutinho, Maria FranciscaSantos, Juliana I.Gonçalves, MarianaGaspar, PauloAlves, Sandra2022-07-102022-07-102021-09http://hdl.handle.net/10400.18/8197About Skipping the Pathology in Rare Diseases: Antisense exon-skipping therapy for Mucolipidosis type IIengDoenças GenéticasGenética HumanaMucolipidosis type IIRNA TherapeuticsLysosomal Storage DisordersExon SkippingOligonucleotide-based TherapiesSkipping the Pathology in Rare Diseases: Antisense exon-skipping therapy for Mucolipidosis type IIconference object