Featured Research

from universities, journals, and other organizations

Mighty Mice Are Less Susceptible To Muscular Dystrophy Gene's Effects

Date:
November 26, 2002
Source:
Johns Hopkins Medical Institutions
Summary:
The Johns Hopkins scientists who first discovered that knocking out a particular muscle gene results in "mighty mice" now report that it also softens the effects of a genetic mutation that causes muscular dystrophy.

The Johns Hopkins scientists who first discovered that knocking out a particular muscle gene results in "mighty mice" now report that it also softens the effects of a genetic mutation that causes muscular dystrophy.

Related Articles


The findings, scheduled for the December issue of the Annals of Neurology and currently online, build support for the idea that blocking the activity of that gene, known as myostatin, may one day help treat humans with degenerative muscle diseases.

Working with mice carrying the genetic mutation that causes Duchenne muscular dystrophy in humans, the scientists discovered that mice without the gene for myostatin had less physical damage to their muscles and were stronger than other mice with the Duchenne mutation.

"'Knocking out' the myostatin gene isn't possible for treating patients, but blocking the myostatin protein might be," says senior investigator Se-Jin Lee, M.D., Ph.D., professor of molecular biology and genetics at Johns Hopkins School of Medicine. "However, myostatin still needs to be studied in people to see if it has the same role in our muscles as it has in mice."

The researchers caution that, even if myostatin does limit muscle growth in people, blocking it would not cure muscular dystrophy or any other degenerative muscle condition because the underlying cause of disease would be unchanged.

"However, increasing muscle mass and strength by blocking myostatin could conceivably delay progression or improve quality of life," notes first author Kathryn Wagner, M.D., Ph.D., assistant professor of neurology at Hopkins.

The Hopkins team bred mice without the myostatin gene with mice carrying the genetic mutation that causes Duchenne muscular dystrophy in humans. Muscular dystrophy mice completely lacking myostatin were more muscular and stronger than those with myostatin at 3, 6 and 9 months of age, the researchers report. Perhaps most importantly, their muscle tissue appeared to be healthier.

Duchenne muscular dystrophy is the most common muscular dystrophy and the most common inherited lethal disease of childhood, affecting 1 in 3,500 live male births. (The genetic mutation that causes it is found on the X chromosome, and so is "covered up" in girls, who have two X chromosomes.) There's no good treatment at this time, and few patients survive into adulthood.

Early in the disease in humans, the regenerative capacity of stem cells in muscle, known as satellite cells, keep up with the damage, but eventually the damaging factors win. The result is not just loss of muscle, but also its replacement with non-muscle tissues, essentially scar tissue and fat.

This scarring process, called fibrosis, is also seen in mice with the muscular dystrophy-causing mutation. The Hopkins team reports that loss of myostatin function significantly reduced the amount of fibrosis, suggesting that the muscle regenerative process was improved.

The Hopkins scientists hope to unravel the mechanism of muscle regeneration in mice with and without myostatin, possibly revealing even better targets for improving the process. They also plan to use special genetic manipulations to turn off the myostatin gene in adult mice, rather than at conception, to see if losing myostatin later in the course of muscular dystrophy is also beneficial.

Authors on the study are Wagner, Lee, Alexandra McPherron and Nicole Winik, all of The Johns Hopkins University School of Medicine. Funding was provided by the National Institutes of Health, the Duchenne Parent Project, and the Muscular Dystrophy Association.

Myostatin was licensed by The Johns Hopkins University to MetaMorphix, Inc., and sublicensed to Wyeth Pharmaceuticals, Inc. Lee and McPherron are entitled to a share of sales royalty received by the University from sales of this factor. Lee, McPherron and the University own MetaMorphix stock, which is subject to certain restrictions under University policy. Lee is a paid consultant to MetaMorphix. The terms of these arrangements are being managed by the University in accordance with its conflict of interest policies.

###

Related Web site:

http://www3.interscience.wiley.com/cgi-bin/abstract/99519627/START


Story Source:

The above story is based on materials provided by Johns Hopkins Medical Institutions. Note: Materials may be edited for content and length.


Cite This Page:

Johns Hopkins Medical Institutions. "Mighty Mice Are Less Susceptible To Muscular Dystrophy Gene's Effects." ScienceDaily. ScienceDaily, 26 November 2002. <www.sciencedaily.com/releases/2002/11/021126072241.htm>.
Johns Hopkins Medical Institutions. (2002, November 26). Mighty Mice Are Less Susceptible To Muscular Dystrophy Gene's Effects. ScienceDaily. Retrieved December 21, 2014 from www.sciencedaily.com/releases/2002/11/021126072241.htm
Johns Hopkins Medical Institutions. "Mighty Mice Are Less Susceptible To Muscular Dystrophy Gene's Effects." ScienceDaily. www.sciencedaily.com/releases/2002/11/021126072241.htm (accessed December 21, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, December 21, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

The Best Tips to Curb Holiday Carbs

The Best Tips to Curb Holiday Carbs

Buzz60 (Dec. 19, 2014) It's hard to resist those delicious but fattening carbs we all crave during the winter months, but there are some ways to stay satisfied without consuming the extra calories. Vanessa Freeman (@VanessaFreeTV) has the details. Video provided by Buzz60
Powered by NewsLook.com
Sierra Leone Bikers Spread the Message to Fight Ebola

Sierra Leone Bikers Spread the Message to Fight Ebola

AFP (Dec. 19, 2014) More than 100 motorcyclists hit the road to spread awareness messages about Ebola. Nearly 7,000 people have now died from the virus, almost all of them in west Africa, according to the World Health Organization. Video provided by AFP
Powered by NewsLook.com
Researchers Test Colombian Village With High Alzheimer's Rates

Researchers Test Colombian Village With High Alzheimer's Rates

AFP (Dec. 19, 2014) In Yarumal, a village in N. Colombia, Alzheimer's has ravaged a disproportionately large number of families. A genetic "curse" that may pave the way for research on how to treat the disease that claims a new victim every four seconds. Duration: 02:42 Video provided by AFP
Powered by NewsLook.com
The Best Protein-Filled Foods to Energize You for the New Year

The Best Protein-Filled Foods to Energize You for the New Year

Buzz60 (Dec. 19, 2014) The new year is coming and nothing will energize you more for 2015 than protein-filled foods. Fitness and nutrition expert John Basedow (@JohnBasedow) gives his favorite high protein foods that will help you build muscle, lose fat and have endless energy. Video provided by Buzz60
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