Tuesday, April 24, 2007

Amazing potential treatment for genetic diseases

Each cell in the body must make thousands of protein molecules to function properly. From start to finish, the protein molecules must be correct and complete.

Thousands of genetic diseases are caused by mutations that produce an erroneous stop signal in the production process of just one type of protein molecule in a cell. The cell needs to make the entire protein molecule, but comes to an erroneous sequence that tells it to stop prematurely, and so it makes just the beginning part of the protein molecule, which is useless. Since one type of protein molecule performs a specific job in a cell, that job is not done, resulting in disease.

Suppose there were a way to make the cell ignore the erroneous stop signal. The cell would then produce the complete protein molecule, and it could then do its specific job in the cell. The disease should theoretically be greatly relieved. And it seems that one drug may do just that for some fraction of thousands of genetic diseases!

http://www.eurekalert.org/pub_releases/2007-04/uops-fdo041807.php

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