Description: Cystic fibrosis is characterized by defects in salt and water transport in a variety of epithelial tissues. The basis of the defect is the gene product termed the cystic fibrosis transmembrane regulator protein (CFTR). The CFTR has been shown to be a chloride channel. The present invention relates to a method of increasing the permeability of epithelial cells to chloride ions in a subject, for example, in treating cystic fibrosis.
Specifically, the invention relates to methodology for the correction of defective chloride transport by increasing the salt and water flux in diseased tissues to levels closer to those found in normal tissues. This treatment will reduce life-threatening complications frequently found in diseases such as cystic fibrosis. The present invention satisfies this need by providing methods and compounds that can therapeutically relieve both the cause of the manifestations of cystic fibrosis, as well as the manifestations themselves.
When appropriately applied, these compounds can correct defective chloride transport, increasing the salt and water flux in diseased tissues to levels closer to those of normal tissues thus reducing life-threatening complications.
There are pathological conditions and disease states other than cystic fibrosis that might also benefit from treatment with compounds that increase salt and water permeability. These include brain swelling, renal disease, and heart disease. |
Issued US Patent #6,159,968 entitled "Activation of chloride channels for correction of defective chloride transport"
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