Linked Life Data

21397500

Source:http://linkedlifedata.com/resource/pubmed/id/21397500

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
8
pubmed:dateCreated
2011-4-4
pubmed:abstractText
Nephropathic cystinosis is a rare autosomal recessive disease characterised by raised intracellular levels of the amino acid, cystine. If untreated, the disease, progressively deteriorates towards end stage renal disease (ESRD) at the end of the first decade. The disease is caused by a defect in the lysosomal transport mechanism for cystine. The treatment of choice is the aminothiol cysteamine which acts as a lysine mimic. However, cysteamine possesses an offensive taste and smell and irritates the gastrointestinal tract leading to nausea and vomiting following administration. Furthermore, the rapid metabolism of cysteamine requires oral administration every 6 h for life, in consequence, the patient compliance is poor. As part of our continuing work to obtain new pro-drugs for the treatment of this genetic disease, we have synthesised a folate derivative of cystamine, the disulfide derivative of cysteamine. This new pro-drug was non cytotoxic, showed greater ability to deplete intralysosomal cystine than the current treatment, and, in fact has been the most effective reducer of intralysosomal cystine discovered in our laboratories to date.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Apr
pubmed:issn
1464-3405
pubmed:author
pubmed:copyrightInfo
Copyright © 2011 Elsevier Ltd. All rights reserved.
pubmed:issnType
Electronic
pubmed:day
15
pubmed:volume
21
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
2502-4
pubmed:dateRevised
2011-11-17
pubmed:meshHeading
pubmed:year
2011
pubmed:articleTitle
Folate pro-drug of cystamine as an enhanced treatment for nephropathic cystinosis.
pubmed:affiliation
School of Pharmacy and Life Sciences, Robert Gordon University, Aberdeen AB10 1FR, UK. ziad.omran@nottingham.ac.uk
pubmed:publicationType
Journal Article, Research Support, Non-U.S. Gov't