A team of researchers at the Stanford University School ofMedicine and the University of California, San Francisco, mayhave found a way to bypass the genetic defect of cystic fibrosis(CF).
CF, the most common lethal genetic disease in whites, resultsfrom a mutation in a lung cell protein called the CFtransmembrane conductance regulator (CFTR). Reports inFebruary issues of Science and Cell suggested that CFTRstraddles the cell membrane and acts as a chloride channel. Theflow of chloride into cells impacts a broad range of cellactivities, including the degree of hydration of mucus. Cyclic-AMP-dependent protein kinase and protein kinase C, enzymesthat add phosphate molecules to proteins, normally activateCFTR and control the flow of chloride.
Mutant CFTR is unable to regulate cellular chloride fluxproperly, at least partially because the protein kinases areunable to activate the channel. This results in an array ofcomplications leading to chronic lung infections and death.
The California researchers reported in the Feb. 28 issue ofNature that a subset of protein kinases, termedcalcium/calmodulin-dependent kinases, are able to activate themutant CFTR via a pathway that circumvents the gene defect.The team said it believes that this differential activation of theCFTR can be used to design compounds that specifically activateCFTR in cystic fibrosis patients and normalize chloride flow.
-- Cynthia Robbins-Roth, Ph.D. BioVenture View
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