ALS Association-funded Study Identifies Potential Therapeutic Target In RNA Enzyme
WASHINGTON, Oct. 28, 2012 /PRNewswire-USNewswire/ --A study funded in part by The ALS Association has revealed a potential new target for therapy to treat amyotrophic lateral sclerosis (ALS) or Lou Gehrig's Disease. This new study, published today in Nature Genetics, shows that lowering the level of a cellular enzyme reduces the disease-causing activity of a major ALS-related protein called TDP-43.
Most people with ALS develop clumps of excess TDP-43 protein within their motor neurons, nerve cells that control movement. The protein clumps, or aggregates, are believed to be toxic to the neurons, through unknown mechanisms. ALS occurs when motor neurons die.
"This finding shows us there may be a variety of ways to target the excess TDP-43 seen in most people with ALS," said Lucie Bruijn, Ph.D., Chief Scientist for The ALS Association. "That is encouraging news for the development of new therapies."
The new study was conducted by Maria Armakola, a graduate student at Stanford University in Palo Alto, Calif., and Matthew Higgins, Ph.D., of the Gladstone Institute of Cardiovascular Disease in San Francisco, Calif., under the direction of Aaron Gitler, Ph.D., of Stanford and Robert Farese, Jr., M.D., of the Gladstone Institute.The study was conducted in yeast cells at risk for dying from TDP-43 aggregates or clumps. The researchers found that deletion of the cellular enzyme called the "RNA lariat debranching enzyme," rescued the at-risk cells. The enzyme normally destroys excess RNA. Without the enzyme, cells accumulate small pieces of RNA, which, the researchers suggest, may act as "decoys" to trap excess TDP-43, thus preventing it from aggregating and causing toxicity. The same protective effect was seen in rodent cells, an encouraging sign that a similar strategy may be effective in human cells.
Select the service that is right for you!COMPARE ALL SERVICES
Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.
- $2.5+ million portfolio
- Large-cap and dividend focus
- Intraday trade alerts from Cramer
- Weekly roundups
Access the tool that DOMINATES the Russell 2000 and the S&P 500.
- Buy, hold, or sell recommendations for over 4,300 stocks
- Unlimited research reports on your favorite stocks
- A custom stock screener
- Upgrade/downgrade alerts
Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.
- Diversified model portfolio of dividend stocks
- Alerts when market news affect the portfolio
- Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.
- Real Money + Doug Kass Plus 15 more Wall Street Pros
- Intraday commentary & news
- Ultra-actionable trading ideas
Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.
- 100+ monthly options trading ideas
- Actionable options commentary & news
- Real-time trading community
- Options TV