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USE OF GUANABENZ FOR THE TREATMENT OF CYSTIC FIBROSIS |
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Principal Investigator : F. Becq
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TECHNICAL DESCRIPTION
Cystic Fibrosis Transmembrane conductance Regulator (CFTR) chloride channel is mutated in CF cells. This mutation leads to a mistrafficking of the protein that is retained in the endoplasmic reticulum.
In vitro experiments on CF epithelial nasal cells have shown that Ganabenz acts on the trafficking of CFTR that is retargeted to the plasma membrane of treated cells.
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