New Hope Exists in Treating Inherited Disease by Suppressing DNA Mutations

-Scientists are getting progressively closer to producing drugs that fix the disrupted protein pathaway, drastically improving the treatment of genetic diseases
-The experimental drug Ataluren may help to treat some cystic fibrosis patients. Ataluren also holds promise in treating more than 2,400 different genetic disorders caused by nonsense mutations.
-the key consideration when treating a genetic disease with suppression therapy, is the fraction of the missing protein that must be restored to yield a therapeutic benefit
-An estimated one-third of gene defects responsible for human disease are thought to come from nonsense mutations.
-Ataluren is now being tested in humans for its effectiveness in treating Duchenne/Becker muscular dystrophy, cystic fibrosis, hemophilia A, hemophilia B and other conditions.

Reflection: This article was interesting but kind of hard to get. I had to re-read certain parts of this in order to fully understand the article. I think the just of it is about the experimental drugs Ataluren and how it helps re-build certain proteins that are not made correctly before child birth.

Link: http://www.sciencedaily.com/releases/2010/04/100426131435.htm

Picture: http://whyfiles.org/212elect_ID/images/prescription_bottle.jpg

Citation:Federation of American Societies for Experimental Biology. "New Hope Exists in Treating Inherited Disease by Suppressing DNA Mutations." ScienceDaily 26 April 2010. 17 May 2010 .