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Cysteamine is a cystine depleting agent used to treat the effects of cystinosis. Cystinosis is a rare disease caused by mutations in the CTNS gene that encodes for cystinosin, a protein responsible for transporting cystine out of the cell lysosome.
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Cysteamine, as a part of CoA plays a crucial role in metabolism by facilitating thioester formation. However, as a free entity, cysteamine is largely absent from metabolism. By contrast, supraphysiological amounts of this simple aminothiol have profound and selective effects of physiology and certain diseases.