Featured Research

from universities, journals, and other organizations

Treatment In Mice Proves Effective Against Duchenne Muscular Dystrophy

Date:
March 21, 2001
Source:
University Of Illinois At Urbana-Champaign
Summary:
Mice carrying the same gene deficiencies as humans with Duchenne muscular dystrophy experienced dramatic improvements in both their physical condition and life span following an experimental treatment by researchers at the University of Illinois.

CHAMPAIGN, Ill. — Mice carrying the same gene deficiencies as humans with Duchenne muscular dystrophy experienced dramatic improvements in both their physical condition and life span following an experimental treatment by researchers at the University of Illinois.

By enhancing the production of a naturally occurring molecule on muscle tissue, the scientists reduced muscle-related problems and increased by three-fold the lifetimes of affected mice. Their work appears in the March 19 issue of the Journal of Cell Biology.

The work suggests that a gene therapy or a pharmaceutical approach targeting the molecule may be possible for human treatment, said Stephen J. Kaufman, a professor of cell and structural biology and of the College of Medicine. "The implications are that you could do gene therapy with an integrin chain to treat a muscular dystrophy that’s caused by a membrane protein deficiency," he said. "Or you could chemically stimulate integrin chain production from the patient’s existing integrin chain genes."

Kaufman’s lab discovered the molecule in question – the alpha 7 integrin – in 1985. A deficiency of this molecule exists in several forms of congenital muscular dystrophy. Conversely, Kaufman and his colleagues found that more of the integrin is present in Duchenne patients. These patients fail to produce another protein, dystrophin, which muscles also require for structural and functional integrity.

This discovery led to the idea that excess integrin may compensate for the lack of dystrophin and another similar protein, utrophin. To test their hypothesis, Kaufman’s team used mice that did not produce dystrophin or utrophin, and they engineered them to produce even more of the alpha 7 integrin protein. Untreated mice developed debilitating muscular dystrophy, suffered severe weight loss and 50 percent died before reaching 12 weeks of age. Mice with enhanced alpha integrin production did not suffer severe muscular problems, maintained good mobility and lived to an average age of 38 weeks.

Duchenne muscular dystrophy, caused by a recessive genetic defect, affects one in 3,300 males. The disease usually begins in early childhood and often is fatal by age 30. It is the most prevalent of the muscular dystrophy family of neuromuscular diseases. Patients with Becker, limb girdle and other muscular dystrophies also might benefit from the approach used in the study, Kaufman said.

"The potential exists to enhance the expression of the endogenous normal alpha 7 integrin gene, or extend the lifetime of the alpha 7 protein," he said. "This would even avoid the need for what we think of as classical gene therapy."

Co-authors with Kaufman on the study were departmental colleague Dean J. Burkin, graduate student Gregory Q. Wallace and former graduate student Kimberly J. Nicol, all of the UI, and David J. Kaufman of the National Cancer Institute in Bethesda, Md. The Muscular Dystrophy Association and National Institutes of Health supported the research.


Story Source:

The above story is based on materials provided by University Of Illinois At Urbana-Champaign. Note: Materials may be edited for content and length.


Cite This Page:

University Of Illinois At Urbana-Champaign. "Treatment In Mice Proves Effective Against Duchenne Muscular Dystrophy." ScienceDaily. ScienceDaily, 21 March 2001. <www.sciencedaily.com/releases/2001/03/010320074741.htm>.
University Of Illinois At Urbana-Champaign. (2001, March 21). Treatment In Mice Proves Effective Against Duchenne Muscular Dystrophy. ScienceDaily. Retrieved September 15, 2014 from www.sciencedaily.com/releases/2001/03/010320074741.htm
University Of Illinois At Urbana-Champaign. "Treatment In Mice Proves Effective Against Duchenne Muscular Dystrophy." ScienceDaily. www.sciencedaily.com/releases/2001/03/010320074741.htm (accessed September 15, 2014).

Share This



More Health & Medicine News

Monday, September 15, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

EU Ministers and Experts Meet to Discuss Ebola Reponse

EU Ministers and Experts Meet to Discuss Ebola Reponse

AFP (Sep. 15, 2014) The European Commission met on Monday to coordinate aid that the EU can offer to African countries affected by the Ebola outbreak. Duration: 00:58 Video provided by AFP
Powered by NewsLook.com
Despite The Risks, Antibiotics Still Overprescribed For Kids

Despite The Risks, Antibiotics Still Overprescribed For Kids

Newsy (Sep. 15, 2014) A new study finds children are prescribed antibiotics twice as often as is necessary. Video provided by Newsy
Powered by NewsLook.com
FDA Eyes Skin Shocks Used at Mass. School

FDA Eyes Skin Shocks Used at Mass. School

AP (Sep. 15, 2014) The FDA is considering whether to ban devices used by the Judge Rotenberg Educational Center in Canton, Massachusetts, the only place in the country known to use electrical skin shocks as aversive conditioning for aggressive patients. (Sept. 15) Video provided by AP
Powered by NewsLook.com
Respiratory Virus Spreads To Northeast, Now In 21 States

Respiratory Virus Spreads To Northeast, Now In 21 States

Newsy (Sep. 14, 2014) The respiratory virus Enterovirus D68, which targets children, has spread from the Midwest to 21 states. 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