Featured Research

from universities, journals, and other organizations

Stem Cell-based Therapy May Be Able To Treat Muscular Dystrophy

Date:
February 25, 2008
Source:
Nature Medicine
Summary:
A new way to manipulate human embryonic stem cells offers hope for an eventual cell-based therapy to treat muscular dystrophies. Muscular dystrophies, such as Duchenne's muscular dystrophy (DMD), are caused by genetic mutations that lead to a loss of expression of dystrophin, a key structural protein of muscle cells, which results in cell dysfunction. When this occurs the cells can no longer regenerate after injury, resulting in progressive muscle weakness and eventual death. One hope for therapy has been to replenish these defective cells with ESCs that produce normal dystrophin.

A new way to manipulate embryonic stem cells (ESCs) offers hope for an eventual cell-based therapy to treat muscular dystrophies.

Muscular dystrophies, such as Duchenne’s muscular dystrophy (DMD), are caused by genetic mutations that lead to a loss of expression of dystrophin, a key structural protein of muscle cells, which results in cell dysfunction. When this occurs the cells can no longer regenerate after injury, resulting in progressive muscle weakness and eventual death. One hope for therapy has been to replenish these defective cells with ESCs that produce normal dystrophin. However, this approach has been hampered by an inability to get ESCs to form muscle cells at appreciable levels.

Rita Perlingeiro and colleagues have overcome this hurdle and show functional recovery after injection of ESCs into a mouse model of DMD. The key to their success was to take advantage of the facts that almost all skeletal muscles have a similar embryonic origin and that muscle development depends on the transcription factor Pax3.

As ESCs grown in a culture dish are not exposed to the embryonic environmental milieu that induces muscle differentiation, it might be possible to bypass this requirement by directly expressing Pax3, which orchestrates muscle development in the embryo, in ESCs through genetic manipulations. This manipulation allows a muscle progenitor cell population to arise and in sufficient quantities to then use therapeutically in mice.

The team was able to deliver these cells through the circulation, targeting many more muscle locations than by intramuscular injection, resulting in significantly improved muscle function. Finally, the use of the isolated muscle progenitor cells, owing to their partially differentiated state, did not result in tumor formation, which has previously hampered the therapeutic use of ESCs.

While the genetic manipulation of the ESCs disallows this technique to be used in the clinic at this point, future studies may point to ways of inducing Pax3 expression in ESCs without the need of genetically modifying the cells.

Journal reference: Radbod Darabi, Kimberly Gehlbach, Robert M Bachoo, Shwetha Kamath, Mitsujiro Osawa, Kristine E Kamm, Michael Kyba & Rita C R Perlingeiro. Functional skeletal muscle regeneration from differentiating embryonic stem cells. Published online: Nature Medicine. 20 January 2008. pp 134 - 143. doi 10.1038/nm1705


Story Source:

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


Cite This Page:

Nature Medicine. "Stem Cell-based Therapy May Be Able To Treat Muscular Dystrophy." ScienceDaily. ScienceDaily, 25 February 2008. <www.sciencedaily.com/releases/2008/02/080224141733.htm>.
Nature Medicine. (2008, February 25). Stem Cell-based Therapy May Be Able To Treat Muscular Dystrophy. ScienceDaily. Retrieved April 20, 2014 from www.sciencedaily.com/releases/2008/02/080224141733.htm
Nature Medicine. "Stem Cell-based Therapy May Be Able To Treat Muscular Dystrophy." ScienceDaily. www.sciencedaily.com/releases/2008/02/080224141733.htm (accessed April 20, 2014).

Share This



More Health & Medicine News

Sunday, April 20, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Nine-Month-Old Baby Can't Open His Mouth

Nine-Month-Old Baby Can't Open His Mouth

Newsy (Apr. 19, 2014) Nine-month-old Wyatt Scott was born with a rare disorder called congenital trismus, which prevents him from opening his mouth. Video provided by Newsy
Powered by NewsLook.com
'Holy Grail' Of Weight Loss? New Find Could Be It

'Holy Grail' Of Weight Loss? New Find Could Be It

Newsy (Apr. 18, 2014) In a potential breakthrough for future obesity treatments, scientists have used MRI scans to pinpoint brown fat in a living adult for the first time. Video provided by Newsy
Powered by NewsLook.com
Little Progress Made In Fighting Food Poisoning, CDC Says

Little Progress Made In Fighting Food Poisoning, CDC Says

Newsy (Apr. 18, 2014) A new report shows rates of two foodborne infections increased in the U.S. in recent years, while salmonella actually dropped 9 percent. Video provided by Newsy
Powered by NewsLook.com
Scientists Create Stem Cells From Adult Skin Cells

Scientists Create Stem Cells From Adult Skin Cells

Newsy (Apr. 17, 2014) The breakthrough could mean a cure for some serious diseases and even the possibility of human cloning, but it's all still a way off. 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