Featured Research

from universities, journals, and other organizations

Enzyme Halts Muscle Waste In Mouse Models Of Most-Common Childhood Muscular Dystrophy

Date:
April 16, 2002
Source:
University Of California - San Diego
Summary:
The muscle destruction associated with Duchenne muscular dystrophy (DMD), the most common childhood form of muscular dystrophy, is halted in mice when supplemental amounts of a naturally occurring enzyme are added to the skeletal muscle.

The muscle destruction associated with Duchenne muscular dystrophy (DMD), the most common childhood form of muscular dystrophy, is halted in mice when supplemental amounts of a naturally occurring enzyme are added to the skeletal muscle.

These results from researchers at the University of California, San Diego (UCSD) School of Medicine are published in the April 16, 2002 issue of the journal Proceedings of the National Academy of Sciences.

Muscle wasting associated with DMD was inhibited after the UCSD team added an enzyme called CT GalNAc transferase to skeletal muscles in mice bred to develop DMD. Normally, CT GalNAc transferase is expressed in another area of the muscle, the neuromuscular junction, where nerves send impulses to muscle fiber. The UCSD team was able to re-position the enzyme so that it was available in the DMD-vulnerable skeletal muscle, which is the structural tissue that supports body movement.

“We hope this enzyme can eventually be used as a therapy for Duchenne muscular dystrophy,” said Paul Martin, Ph.D., UCSD assistant professor of neurosciences, a member of the UCSD Glycobiology Training and Research Center, and the study’s senior author. “It has the potential for managing the disease, much like we manage diabetes with insulin medication or injections.”

The disease strikes one in 5,000 children, almost exclusively boys, before they reach the age of 6. DMD frequently leaves its victims wheelchair-bound by age 12 and most DMD children die in their early 20s. The disease is caused by a mutation in a protein called dystrophin, which helps anchor muscle fibers to the connective tissue surrounding them.

“Losing dystrophin is like losing the foundation of your house,” Martin said. “Without that foundation, the house falls apart. Without dystrophin, muscles affected by DMD become damaged and break.”

Martin discovered the enzyme as he looked for other proteins that might compensate for the disabled dystrophin in DMD patients. Normally, CT GalNAc transferase acts as a chemical stimulant to generate scaffold-like tissue at the neuromuscular junction. In research reported earlier this year in the journal Developmental Biology (242, 58-73), Martin and his team described the role of CT GalNAc transferase and their development of mice (called CT mice) that had the enzyme available in skeletal muscles, as well as the neuromuscular junction. These mice did not develop the DMD muscle wasting.

In their most recent experiments, the Martin team bred the CT mice to mice that lacked the dystrophin gene (called mdx mice). While mdx mice developed muscle wasting indicative of DMD, the disease did not develop in the new breed of CT-mdx mice.

The next steps for Martin’s lab will be to refine the science behind the use of CT GalNAc transferase for DMD. Although he hesitates to say when a therapy might be available for human clinical trials, Martin notes that it may require a pharmaceutical company that is willing to invest in drug development.

“There are two schools of thought about how to develop a therapy,” he said. “One option is gene therapy. Although a small amount of enzyme would have a big effect, the mechanics of administering the gene to all muscles in the body would not be an easy task. We’re more excited about the possibility of creating an easy-to-administer oral medication that could globally stimulate this enzyme in skeletal muscles.”

In addition to Martin, additional authors of the study were Holly H. Nguyen, a second year UCSD medical student, and UCSD researchers Vianney Jayasinha, Bing Zia, and Kwame Hoyte.

The work was funded by grants from the Multiple Dystrophy Association, the March of Dimes, and the National Institutes of Health.


Story Source:

The above story is based on materials provided by University Of California - San Diego. Note: Materials may be edited for content and length.


Cite This Page:

University Of California - San Diego. "Enzyme Halts Muscle Waste In Mouse Models Of Most-Common Childhood Muscular Dystrophy." ScienceDaily. ScienceDaily, 16 April 2002. <www.sciencedaily.com/releases/2002/04/020416074041.htm>.
University Of California - San Diego. (2002, April 16). Enzyme Halts Muscle Waste In Mouse Models Of Most-Common Childhood Muscular Dystrophy. ScienceDaily. Retrieved July 25, 2014 from www.sciencedaily.com/releases/2002/04/020416074041.htm
University Of California - San Diego. "Enzyme Halts Muscle Waste In Mouse Models Of Most-Common Childhood Muscular Dystrophy." ScienceDaily. www.sciencedaily.com/releases/2002/04/020416074041.htm (accessed July 25, 2014).

Share This




More Health & Medicine News

Friday, July 25, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

New Painkiller Designed To Discourage Abuse: Will It Work?

New Painkiller Designed To Discourage Abuse: Will It Work?

Newsy (July 24, 2014) The FDA approved Targiniq ER on Wednesday, a painkiller designed to keep users from abusing it. Like any new medication, however, it has doubters. Video provided by Newsy
Powered by NewsLook.com
Doctor At Forefront Of Fighting Ebola Outbreak Gets Ebola

Doctor At Forefront Of Fighting Ebola Outbreak Gets Ebola

Newsy (July 24, 2014) Sheik Umar Khan has treated many of the people infected in the Ebola outbreak, and now he's become one of them. Video provided by Newsy
Powered by NewsLook.com
Condemned Man's US Execution Takes Nearly Two Hours

Condemned Man's US Execution Takes Nearly Two Hours

AFP (July 24, 2014) America's death penalty debate raged Thursday after it took nearly two hours for Arizona to execute a prisoner who lost a Supreme Court battle challenging the experimental lethal drug cocktail. Duration: 00:55 Video provided by AFP
Powered by NewsLook.com
Can Watching TV Make You Feel Like A Failure?

Can Watching TV Make You Feel Like A Failure?

Newsy (July 24, 2014) A study by German researchers claims watching TV while you're stressed out can make you feel guilty and like a failure. Video provided by Newsy
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