Article

Use of human iPSCs and kidney organoids to develop a cysteamine/mTOR inhibition combination therapy to treat cystinosis

2019-04-01

Abstract excerpt

Cystinosis is a lysosomal storage disease caused by mutations in CTNS , encoding a cystine transporter, and in its severest form is characterized by cystine accumulation, renal proximal tubule dysfunction and kidney failure. Cystinosis is treated with the cystine-depleting drug cysteamine, however this only slows progression of the disease and there is an urgent need for better treatments. Here, we have generated...

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Literature Corpus work: 925416d9-981e-5a96-84a7-fe3ef85a7e19
DOI: 10.1101/595264

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