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Kidney transplantation and cystine-depleting therapy with oral and topical cysteamine have transformed cystinosis from a universally fatal childhood disorder into a treatable chronic disease, with many adults achieving normal lives

Final concentrations of K105X and E170X mutants were 10 nM to 1.5 M and 520 nM, respectively

These findings suggest that ASCT2 is a novel therapeutic target for CRC

Adding further complexity, regional heterogeneity in microglial phenotypes, such as differences between striatal and cortical microglia in PD, can influence treatment responses and must be carefully considered in the design of targeted therapies