Featured Research

from universities, journals, and other organizations

Toward An Effective Treatment For Spinal Muscular Atrophy

Date:
October 14, 2008
Source:
American Chemical Society
Summary:
Scientists are reporting a key advance toward developing the first effective drug treatment for spinal muscular atrophy (SMA), a genetic disease that involves motor neuron loss and occurs in 1 out of every 6,000 births. SMA is the leading cause of hereditary infant death in the United States.

Scientists are reporting a key advance toward developing the first effective drug treatment for spinal muscular atrophy (SMA), a genetic disease that involves motor neuron loss and occurs in 1 out of every 6,000 births. SMA is the leading cause of hereditary infant death in the United States.

Mark E. Gurney, Jill Jarecki, and colleagues note that SMA is caused by a defective gene, SMN1, which fails to produce sufficient amounts of a key protein, called SMN (survival motor neuron), needed for normal motor neuron development. Scientists have screened more than 550,000 compounds in the search for a new SMA drug. Recent research pointed to a group of compounds called C5-quinazolines that can boost SMN2 activity, a uniquely existing back-up gene for SMN1. In doing so, they showed promise for treating SMA by producing increased amounts of the needed protein.

In the new study, researchers identified exactly how these promising compounds work, a key step in moving forward toward medical use. They found that the substance targets a normal cellular protein, DcpS, involved in mRNA metabolism whose inhibition causes increased SMN expression. The finding could help guide the development of the first effective drugs for treating SMA and also lead to second generation drugs targeting this enzyme, the researchers say.

"The results outlined in the paper and carried out in collaboration with Families of SMA, deCODE chemistry & biostructures, Invitrogen Corporation, and Rutgers University represent a new understanding of the physiological mechanisms that can increase SMN expression and will allow us to move forward in advancing potential treatments for it, says Jill Jarecki, Ph.D., Research Director at Families of SMA.


Story Source:

The above story is based on materials provided by American Chemical Society. Note: Materials may be edited for content and length.


Journal Reference:

  1. Singh et al. DcpS as a Therapeutic Target for Spinal Muscular Atrophy. ACS Chemical Biology, October 2008 DOI: 10.1021/cb800120t

Cite This Page:

American Chemical Society. "Toward An Effective Treatment For Spinal Muscular Atrophy." ScienceDaily. ScienceDaily, 14 October 2008. <www.sciencedaily.com/releases/2008/10/081013110208.htm>.
American Chemical Society. (2008, October 14). Toward An Effective Treatment For Spinal Muscular Atrophy. ScienceDaily. Retrieved September 19, 2014 from www.sciencedaily.com/releases/2008/10/081013110208.htm
American Chemical Society. "Toward An Effective Treatment For Spinal Muscular Atrophy." ScienceDaily. www.sciencedaily.com/releases/2008/10/081013110208.htm (accessed September 19, 2014).

Share This



More Health & Medicine News

Friday, September 19, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

The Cost of Ebola

The Cost of Ebola

Reuters - Business Video Online (Sep. 18, 2014) As Sierra Leone prepares for a three-day "lockdown" in its latest bid to stem the spread of Ebola, Ciara Lee looks at the financial implications of fighting the largest ever outbreak of the disease. Video provided by Reuters
Powered by NewsLook.com
What HealthKit Bug Means For Your iOS Fitness Apps

What HealthKit Bug Means For Your iOS Fitness Apps

Newsy (Sep. 18, 2014) Apple has delayed the launch of the HealthKit app platform, citing a bug. Video provided by Newsy
Powered by NewsLook.com
U.S. Food Makers Surpass Calorie-Cutting Pledge

U.S. Food Makers Surpass Calorie-Cutting Pledge

Newsy (Sep. 18, 2014) Sixteen large food and beverage companies in the United States that committed to cut calories in their products far surpassed their target. Video provided by Newsy
Powered by NewsLook.com
Residents Vaccinated as Haiti Fights Cholera Epidemic

Residents Vaccinated as Haiti Fights Cholera Epidemic

AFP (Sep. 18, 2014) Haitians receive the second dose of the vaccine against cholera as part of the UN's vaccination campaign. Duration: 00:34 Video provided by AFP
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins