Proteomics and New Therapies for Cystic Fibrosis

The genomics revolution has afforded unparalleled opportunities to understand in detail the pathogenesis of human diseases and design and implement novel, revolutionary therapies. However, the genomics revolution requires that a sequence of "follow-on" technologies be developed.

As one example, it is important to understand the nature of the structure and function of the proteins that have been/will be identified as a function of the genomics revolution and, most importantly, to understand how they function in the context of living organisms. The field of "proteomics" consequently has evolved to provide investigators world-wide the capacity to measure the level of proteins expressed in individual human cells and, most importantly, understand how they interact with other proteins to perform normal cellular functions, and how they may function abnormally in disease processes.

Such knowledge is critical for developing novel therapies for the major lethal disease of white children in the United States, cystic fibrosis. In cystic fibrosis, the affected cells of the lung exhibit abnormalities that reflect the absence of the CF protein's (known as CFTR) function itself, and also the absence of important interactions with other cellular proteins. The identification of these interactions is key to developing therapies to "take up the slack" for missing CFTR-"other" cell protein interactions that are important for cell, organ-level and patient health.
Indeed, it is highly likely that knowledge of the CFTR-"other" protein interactions in lung epithelial cells will allow us to develop a host of novel therapeutic strategies to rid the lungs of CF patients of chronic infections that lead to lung destruction and death.