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.

Related Articles


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 October 30, 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 October 30, 2014).

Share This



More Health & Medicine News

Thursday, October 30, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Mind-Controlled Prosthetic Arm Restores Amputee Dexterity

Mind-Controlled Prosthetic Arm Restores Amputee Dexterity

Reuters - Innovations Video Online (Oct. 29, 2014) A Swedish amputee who became the first person to ever receive a brain controlled prosthetic arm is able to manipulate and handle delicate objects with an unprecedented level of dexterity. The device is connected directly to his bone, nerves and muscles, giving him the ability to control it with his thoughts. Matthew Stock reports. Video provided by Reuters
Powered by NewsLook.com
Google To Use Nanoparticles, Wearables To Detect Disease

Google To Use Nanoparticles, Wearables To Detect Disease

Newsy (Oct. 29, 2014) Google X wants to improve modern medicine with nanoparticles and a wearable device. It's all an attempt to tackle disease detection and prevention. Video provided by Newsy
Powered by NewsLook.com
Can Drinking Milk Lead To Early Death?

Can Drinking Milk Lead To Early Death?

Newsy (Oct. 29, 2014) Researchers in Sweden released a study showing heavy milk drinkers face an increased mortality risk from a variety of causes. Video provided by Newsy
Powered by NewsLook.com
Obama: The US Will Not 'run and Hide' From Ebola

Obama: The US Will Not 'run and Hide' From Ebola

AP (Oct. 29, 2014) Surrounded by health care workers in the White House East Room, President Barack Obama said the U.S. will likely see additional Ebola cases in the weeks ahead. But he said the nation can't seal itself off in the fight against the disease. (Oct. 29) Video provided by AP
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:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

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