Amaral, Olga2020-06-012020-06-012019-10http://hdl.handle.net/10400.18/6892Invited speakerInvited speaker at Med-Lysosomal2019. Following the success of three earlier meetings on rare diseases wherein LSDs were important components, the meeting in London focused on the diagnosis and treatment as well as on new therapeutic modalities already in advanced stages of development of four main Lysosomal Diseases: Gaucher, Fabry, Pompe and MPSs.engHuman GeneticsGene EditingCRISPR/CasCell ModelsDoenças Genéticaslecture