Featured Research

from universities, journals, and other organizations

New Therapy Substitutes Missing Protein In Those With Muscular Dystrophy

Date:
May 27, 2009
Source:
University of Minnesota
Summary:
Researchers have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children.

Researchers at the University of Minnesota Medical School have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children.

Related Articles


In the mouse model, researchers were able to substitute for the missing protein – dystrophin, which forms a key part of the framework that holds muscle tissue together – that results in the disease, effectively repairing weakened muscle tissue.

Researchers injected dystrophic mice with a protein called utrophin – a very close relative of dystrophin – that was modified with a cell-penetrating tag, called TAT.

The study is the first to establish the efficacy and feasibility of the TAT-utrophin-based protein as a viable therapy for the treatment of muscular dystrophy as well as cardiac muscle diseases caused by loss of dystrophin.

The research is published in the May 26, 2009 issue of PLoS Medicine.

"This unique approach can replace the missing protein without the complexities of gene replacement or stem cell approaches," said James Ervasti, Ph.D., principal investigator of the study and a professor in the Department of Biochemistry, Molecular Biology & Biophysics.

Muscular dystrophy causes the muscles in the body to progressively weaken. Duchenne is the most common and severe form of childhood muscular dystrophy. About one of 3,500 boys are born with the crippling disease. Symptoms usually begin in children who are 2 to 3 years-old, most are in a wheelchair by age 12, and many who have the disease pass away by their late teens to early 20s. Current treatment, limited to corticosteroids, are minimally effective and can cause serious side effects.

Research underway to battle muscular dystrophy with gene therapy and stem cell treatment shows promise, but major hurdles must be overcome before these approaches are viable in human patients, Ervasti said.

Delivering treatment to every muscle cell via gene therapy or stem cells is difficult because muscle tissue makes up such a large portion of the human body. Furthermore, the immune system may reject the cell or gene treatment because patients would treat the newly introduced cells or genes as a foreign substance.

Ervasti's method may conquer both of those problems. Upon injection, the TAT-utrophin combination spreads around the entire body efficiently and is able to penetrate the muscle cell wall to substitute for missing dystrophin. Because every cell in the body makes utrophin naturally, TAT-utrophin circumvents immunity issues associated with other therapeutic approaches.

"Our protein replacement approach most directly and simply addresses the cause of Duchenne muscular dystrophy," Ervasti said.

This new method is not a cure for muscular dystrophy. Rather, it would be a therapy most likely administered on a regular basis. If the treatment works in larger animal models and humans, it's most likely researchers would develop a drug for patients. Ervasti is hopeful the therapy can move into human clinical trials within 3 years.

The research was funded by the Muscular Dystrophy Association, the Nash Avery Foundation, Charley's Fund and the Foundation to Eradicate Duchenne.


Story Source:

The above story is based on materials provided by University of Minnesota. Note: Materials may be edited for content and length.


Cite This Page:

University of Minnesota. "New Therapy Substitutes Missing Protein In Those With Muscular Dystrophy." ScienceDaily. ScienceDaily, 27 May 2009. <www.sciencedaily.com/releases/2009/05/090526152713.htm>.
University of Minnesota. (2009, May 27). New Therapy Substitutes Missing Protein In Those With Muscular Dystrophy. ScienceDaily. Retrieved November 23, 2014 from www.sciencedaily.com/releases/2009/05/090526152713.htm
University of Minnesota. "New Therapy Substitutes Missing Protein In Those With Muscular Dystrophy." ScienceDaily. www.sciencedaily.com/releases/2009/05/090526152713.htm (accessed November 23, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, November 23, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

WFP: Ebola Risks Heightened Among Women Throughout Africa

WFP: Ebola Risks Heightened Among Women Throughout Africa

AFP (Nov. 21, 2014) Having children has always been a frightening prospect in Sierra Leone, the world's most dangerous place to give birth, but Ebola has presented an alarming new threat for expectant mothers. Duration: 00:37 Video provided by AFP
Powered by NewsLook.com
Could Your Genes Be The Reason You're Single?

Could Your Genes Be The Reason You're Single?

Newsy (Nov. 21, 2014) Researchers in Beijing discovered a gene called 5-HTA1, and carriers are reportedly 20 percent more likely to be single. Video provided by Newsy
Powered by NewsLook.com
Raw: Paralyzed Marine Walks With Robotic Braces

Raw: Paralyzed Marine Walks With Robotic Braces

AP (Nov. 21, 2014) Marine Corps officials say a special operations officer left paralyzed by a sniper's bullet in Afghanistan walked using robotic leg braces in a ceremony to award him a Bronze Star. (Nov. 21) Video provided by AP
Powered by NewsLook.com
Milestone Birthdays Can Bring Existential Crisis, Study Says

Milestone Birthdays Can Bring Existential Crisis, Study Says

Newsy (Nov. 21, 2014) Researchers find that as people approach new decades in their lives they make bigger life decisions. 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:

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