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Fail-safe system may lead to cures for inherited disorders
Date:9/15/2011

tion only in the appropriate tissues, a highly selective effect can potentially be achieved" said Wilkinson.

"For example, there is a need to come up with better treatments for cystic fibrosisa heritable chronic lung diseasethat is currently being treated in some patients with drugs that act by blocking recognition of the premature stop signal in the mutant CFTR gene," he said. "There has been some success with this approach, but there are concerns with side effects." The finding that NMD is buffered by a tissue-specific regulatory system means that one could design a different type of druga tissue-specific NMD-inhibition drugthat increases the level of the CFTR protein primarily in its main cellular site of action: the lung. "This could potentially increase the efficacy and drastically reduce the side effects of NMD-inhibition drugs" says Wilkinson.


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Contact: Scott LaFee
slafee@ucsd.edu
619-543-6163
University of California - San Diego
Source:Eurekalert

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