Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 2 of 2 for “"ML-SA5"”.
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MODULATION OF TRPML1/TFEB PATHWAY FOR THE TREATMENT OF WILSON DISEASE
… transient receptor potential mucolipin 1 (TRPML1) activates calcineurin, which dephosphorylates TFEB, promoting its nuclear translocation. In this study, we investigated the therapeutic potential of TRPML1/TFEB axis for the treatment of WD. We first assessed the effects of TFEB and TRPML1 …
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Phenotypic characterisation of human iPSC neuronal models of GM2 gangliosidoses
… for these diseases, I trial the use of ML-SA5, a drug shown to induce lysosomal exocytosis in non-neuronal cell types and proposed as a treatment for neurodegenerative lysosomal storage disorders. I show the deleterious effects that this drug has on synaptic signalling, without …