Use of Human Induced Pluripotent Stem Cells and Kidney Organoids To Develop a Cysteamine/mTOR Inhibition Combination Therapy for Cystinosis.
Jennifer A HollywoodAneta J PrzepiorskiRandall F D'SouzaSreevalsan SreebhavanErnst J WolvetangPatrick T HarrisonAlan J DavidsonTeresa M HolmPublished in: Journal of the American Society of Nephrology : JASN (2020)
These observations suggest that combination therapy with a cystine-depleting drug such as cysteamine and an mTOR pathway inhibitor such as everolimus has potential to improve treatment of cystinosis.