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Design, Synthesis and Initial In Vitro Evaluation of Novel Prodrugs for the Treatment of Cystinosis

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Simple amino acid prodrugs of cysteamine and cystamine were synthesized to overcome the problems of palatability and gastrointestinal irritation, while γ-glutamyl prodrugs were synthesized to target the parent drug to γ-glutamyl transpeptidase (GGT). Preliminary cystine-depleting evaluation in vitro suggests further investigation is warranted. Low toxicity of the prodrugs was observed.





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Keywords: Cystinosis; GGT; cystamine; cysteamine; prodrugs

Document Type: Research Article

Affiliations: Sunderland Pharmacy School, University of Sunderland, Sunderland, SR1 3SD, UK.

Publication date: 01 July 2006

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  • Letters in Drug Design & Discovery publishes original letters on all areas of rational drug design and discovery including medicinal chemistry, in-silico drug design, combinatorial chemistry, high-throughput screening, drug targets, and structure-activity relationships. The emphasis will be on publishing quality papers very rapidly. Letters will be processed rapidly by taking full advantage of Internet technology for both the submission and review of manuscripts. The journal is essential reading to all pharmaceutical scientists involved in research in drug design and discovery.
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