Santos, Juliana InêsCoutinho, Maria FranciscaGaspar, PauloAlves, Sandra2019-03-252019-03-252018-03http://hdl.handle.net/10400.18/6288About proof of principle in genetically modulated substrate reduction therapy for Sanfilippo syndrome.engLysosomal Storage DiseasesMucopilysaccharidosesSubstrate Reduction TherapyRNAiDoenças GenéticasGenética HumanaGenetically Modulated Substrate Reduction Therapy for Sanfilippo Syndrome: Proof of Principleconference object