Bakers research on nonsense-mediated mRNA decay not only provides an advanced understanding of an important process in the regulation of gene expression, but may help lead to new therapeutic strategies in the treatment of genetic diseases. Many inherited conditions, including cystic fibrosis, are a consequence of mutations resulting in the recognition of non-functional mRNA and the subsequent elimination by nonsense-mediated mRNA decay. Because cells eliminate the abnormal mRNA, no protein is produced. With genetic diseases, researchers are hypothesizing it might be beneficial for the cell to express the protein, even though it is not completely functional. The rational is it will be better for these patients to have protein of some function rather than no protein at all. Cystic fibrosis clinical trials are currently underway with a goal of producing the partially functional proteins, before the cells natural elimination process takes place. Using Bakers findings, researchers will have a better understanding of how to modulate the recognition of the abnormal mRNAs as to allow the mRNA to remain in the cell and produce the protein.
This finding is an important step in advancing our understanding of mRNA function, said Baker. In addition, it emphasizes the important link between basic and clinical science; the more we understand the basic biological processes that are underway in the cell, the better equipped we are to directly address clinical therapies.
|Contact: Jessica Studeny|
Case Western Reserve University