Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Mighty mice are less susceptible to muscular dystrophy gene’s effects

26.11.2002


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 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.

Media Contact: Joanna Downer 410-614-5105
Email: jdowner1@jhmi.edu

Joanna Downer | EurekAlert!
Further information:
http://www3.interscience.wiley.com/cgi-bin/abstract/99519627/START

More articles from Life Sciences:

nachricht Flow of cerebrospinal fluid regulates neural stem cell division
22.05.2018 | Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt

nachricht Chemists at FAU successfully demonstrate imine hydrogenation with inexpensive main group metal
22.05.2018 | Friedrich-Alexander-Universität Erlangen-Nürnberg

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Explanation for puzzling quantum oscillations has been found

So-called quantum many-body scars allow quantum systems to stay out of equilibrium much longer, explaining experiment | Study published in Nature Physics

Recently, researchers from Harvard and MIT succeeded in trapping a record 53 atoms and individually controlling their quantum state, realizing what is called a...

Im Focus: Dozens of binaries from Milky Way's globular clusters could be detectable by LISA

Next-generation gravitational wave detector in space will complement LIGO on Earth

The historic first detection of gravitational waves from colliding black holes far outside our galaxy opened a new window to understanding the universe. A...

Im Focus: Entangled atoms shine in unison

A team led by Austrian experimental physicist Rainer Blatt has succeeded in characterizing the quantum entanglement of two spatially separated atoms by observing their light emission. This fundamental demonstration could lead to the development of highly sensitive optical gradiometers for the precise measurement of the gravitational field or the earth's magnetic field.

The age of quantum technology has long been heralded. Decades of research into the quantum world have led to the development of methods that make it possible...

Im Focus: Computer-Designed Customized Regenerative Heart Valves

Cardiovascular tissue engineering aims to treat heart disease with prostheses that grow and regenerate. Now, researchers from the University of Zurich, the Technical University Eindhoven and the Charité Berlin have successfully implanted regenerative heart valves, designed with the aid of computer simulations, into sheep for the first time.

Producing living tissue or organs based on human cells is one of the main research fields in regenerative medicine. Tissue engineering, which involves growing...

Im Focus: Light-induced superconductivity under high pressure

A team of scientists of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg investigated optically-induced superconductivity in the alkali-doped fulleride K3C60under high external pressures. This study allowed, on one hand, to uniquely assess the nature of the transient state as a superconducting phase. In addition, it unveiled the possibility to induce superconductivity in K3C60 at temperatures far above the -170 degrees Celsius hypothesized previously, and rather all the way to room temperature. The paper by Cantaluppi et al has been published in Nature Physics.

Unlike ordinary metals, superconductors have the unique capability of transporting electrical currents without any loss. Nowadays, their technological...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

Save the date: Forum European Neuroscience – 07-11 July 2018 in Berlin, Germany

02.05.2018 | Event News

Invitation to the upcoming "Current Topics in Bioinformatics: Big Data in Genomics and Medicine"

13.04.2018 | Event News

Unique scope of UV LED technologies and applications presented in Berlin: ICULTA-2018

12.04.2018 | Event News

 
Latest News

Supersonic waves may help electronics beat the heat

18.05.2018 | Power and Electrical Engineering

Keeping a Close Eye on Ice Loss

18.05.2018 | Information Technology

CrowdWater: An App for Flood Research

18.05.2018 | Information Technology

VideoLinks
Science & Research
Overview of more VideoLinks >>>