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A potential new prodrug for the treatment of cystinosis:Design, synthesis and in-vitro evaluation

McCaughan, Bridgeen, Kay, Graeme, Knott, Rachel M and Cairns, Donald (2008) A potential new prodrug for the treatment of cystinosis:Design, synthesis and in-vitro evaluation. Bioorganic & Medicinal Chemistry Letters, 18 . pp. 1716-1719. [Journal article]

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DOI: 10.1016/j.bmcl.2008.01.039

Abstract

Nephropathic cystinosis is a rare autosomal recessive disease characterised by raised lysosomal levels of cystine in thecells of most organs. The disorder is treated by regular administration of the aminothiol, cysteamine, an odiferous and unpleasanttasting compound that along with its metabolites is excreted in breath and sweat, leading to poor patient compliance. In an attemptto improve patient compliance a series of novel prodrugs has been designed and evaluated as a potential new treatment for nephropathiccystinosis. The first of the prodrugs tested, 3a, was found to decrease the levels of intracellular cystine in cystinotic fibroblasts.This is the first report of a potential new therapeutic treatment for nephropathic cystinosis since the advent of cysteaminebitartrate.

Item Type:Journal article
Faculties and Schools:Faculty of Life and Health Sciences > School of Pharmacy and Pharmaceutical Science
Faculty of Life and Health Sciences
Research Institutes and Groups:Biomedical Sciences Research Institute
Biomedical Sciences Research Institute > Pharmacy & Pharmaceutical Sciences
ID Code:12257
Deposited By: Dr Bridgeen Callan
Deposited On:17 Sep 2013 11:40
Last Modified:09 Dec 2015 10:45

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