This Day On The Street
Continue to site right-arrow
ADVERTISEMENT
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here
TheStreet Open House

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.

1 of 2

Check Out Our Best Services for Investors

Action Alerts PLUS

Jim Cramer and Stephanie Link reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
Stocks Under $10

Jim Cramer's protégé, David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
Try it NOW
Try it NOW
Try it NOW

Check Out Our Best Services for Investors

Dividend Stock Advisor

Jim Cramer's protégé, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Options Profits

Our options trading pros provide over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • Actionable options commentary and news
  • Real-time trading community
Try it NOW
Try it NOW
Try it NOW
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!

Markets

DOW 17,164.95 -251.90 -1.45%
S&P 500 1,994.99 -26.26 -1.30%
NASDAQ 4,635.24 -48.1670 -1.03%

Partners Compare Online Brokers

Free Reports

Free Newsletters from TheStreet

My Subscriptions:

After the Bell

Before the Bell

Booyah! Newsletter

Midday Bell

TheStreet Top 10 Stories

Winners & Losers

Register for Newsletters
Top Rated Stocks Top Rated Funds Top Rated ETFs