Tuesday November 24 2009 | Biotechnology feed | All feeds
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Tuesday November 24 2009 | Biotechnology feed | All feeds
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Novel approaches to cystic fibrosis Cystic fibrosis is the most common genetically inherited disease to affect Caucasians. Major breakthroughs were made in the 1990's resulting in the identification of mutations in the chloride channel CFTR as the cause of disease both in the airway and the intestine as well as other epithelia. However, progress towards an effective treatment has been disappointing. Deletion of three nucleotides from the CFTR gene causes prevents the expression of phenylalanine at position 508 of CFTR and accounts for the majority of cases of cystic fibrosis (D508). This mutation causes a defect in trafficking so that CFTR does not reach the plasma membrane and is instead degraded. S-Nitrosoglutathione (GSNO) is an endogenous bronchodilator, levels of which are reduced in the airways of cystic fibrosis patients. GSNO has recently been shown to increase maturation of CFTR in cystic fibrosis cell lines at physiological concentrations. The ability of S-nitrosoglutathione to direct the DF508-CFTR to the plasma membrane and restore the function of the cAMP-dependent chloride transport in cultured human airway epithelial cells has now been studied. Swedish researchers have used immunocytochemical methodologies to show that GSNO time- and dose-dependently increases apical CFTR. Chloride transport studies showed that this was mirrored by a fourfold increase of cAMP-dependent chloride transport. This data and the fact that endogenous GSNO levels are lower in the airways of CF patients suggests that GSNO may underlie a novel approach to the treatment of cystic fibrosis. December, 2002
Adapted from Andersson et al, Biochem Biophys Res Commun 2002 Sep 27;297(3):552-7 Interested in collaborating with this group? Contact leaddiscovery@bioportfolio.co.uk Projects such as these are overviewed in full DiscoveryDossiers. LeadDiscovery and BioPortfolio aims to provide reliable, insightful analysis on the biotechnology industry. However, this information is provided "as is" and no representations or warranties either express or implied of completeness, accuracy, or of any other nature are made with respect to this information. This information is neither an offer to sell nor a solicitation to buy the securities of any company. This information contains forward-looking statements, which involve risks and uncertainties which may not be listed. The biotechnology industry is an emerging industry and the securities of the companies mentioned in this report have a very high degree of risk and volatility. For this reason, this information is supplied on the condition that the reader will make his or her own determination as to its suitability for any purpose prior to any use of this information. The employees and officers of LeadDiscovery and BioPortfolio may hold positions in some or all of the stocks discussed in this report. This abstract has been produced by LeadDiscovery Ltd. Founded by life scientists for life scientists we aim to help industry identify cutting edge drug discovery options and academic/biotech institutions maximize the potential of their research. Abstracts strictly reflect the opinion of LeadDiscovery's editorial panel. While all reasonable efforts are made to ensure the accuracy of information provided LeadDiscovery and the publisher BioPortfolio, takes no responsibility for incorrect or misleading information. LeadDiscovery is designed for educational and drug development purposes only and is not intended or designed to offer medical advice or advice of any sort, and must not be used for such purpose. The information provided through LeadDiscovery and BioPortfolio should not be used for diagnosing or treating a health problem or a disease and no reliance should be placed on any information contained in this abstract or elsewhere on LeadDiscovery's and BioPortfolio's website. It is not intended to be a substitute for professional care. If you have or suspect you may have a health problem, you should consult your physician or other health care provider. |
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