Stem Cell Therapy Improves Muscle Function in Muscular Dystrophy

by Medindia Content Team on  January 21, 2008 at 1:12 PM Genetics & Stem Cells News
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Stem Cell Therapy Improves Muscle Function in Muscular Dystrophy
A treatment for muscular dystrophy may be within view after US scientists reported Sunday they used stem cell transplants to improve muscle function in mice afflicted with the disease.

This is the first time transplanted embryonic stem cells have been shown to restore defective muscles in mice models of the wasting disease, according to the study.

"We envision eventually developing a stem-cell therapy for humans," said University of Texas molecular biologist Rita Perlingeiro, who led the research.

"These cells can be transplanted into the muscle, and they cause muscle regeneration," she said, adding that any therapy for humans was still years away.

Muscular dystrophy is an umbrella term covering nearly a dozen genetic, degenerative diseases that primarily affect muscles.

The condition, which afflicts mainly males, is caused by an absence of dystrophin, a protein essential for keeping muscle cells intact and alive.

The most common childhood form, Duchenne muscular dystrophy, shows up at age three or four. Its victims rarely live into their thirties.

In the study, researchers report on a new technique which allowed them to coax embryonic mouse cells into becoming muscle cells that were injected directly into rodents with the Duchenne form of the disease.

"The problem has been that embryonic cells make everything," said Perlingeiro. "The trick is to pull out only the one type you want."

In order to do this the researchers manipulated a gene called Pax3 that is active in the earliest stages of stem cell development -- before the cells become, in other words, blood, bone, muscle or other specialised tissue in the body.

In a first set of experiments, the results were mixed.

"We grew muscle in vitro -- in the Petri dish -- but when we injected the cells into the mice there were still some left-over undifferentiated cells, and these caused tumours," Perlingeiro told AFP.

To filter out all the unwanted cells, the researchers used fluorescent dyes that made it easier to identify the different types of cells.

This time, when the purified batch of muscle cells was injected into the hind legs of mice with muscular dystrophy, the cells penetrated deep into the muscle, an indication that they were growing and reproducing as intended.

Many of the muscle fibres also contained the all-important dystrophin that is lacking in muscular dystrophy patients.

In tests measuring muscle development, the muscles contracted with three times as much force as control mice who had the disease but had not been given the treatment. The results were in fact closer to normal mice, the study reported.

And even after three months, there were no tumours.

The next step, said Perlingeiro, is to extend the same technique to humans. "We should be able to use the same strategy," she said.

Combining these findings with recent breakthroughs in the generation of pluripotent stem cells from adult human skin -- thus avoiding the ethical quandaries of using human embryo stem cells -- would hasten the process, she said.

Source: AFP
LIN/S

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rajeshjipnp Saturday, March 24, 2012

mY doughter IS SUFFEREING THE MUSCULAR DYSTROPHY FOR THE LAST 13 YEARS KINLDY TELL US ANY MEIDCINE or tretment in home base FOR THIS . ANY solution FOUND PLS LET US KNOW.

nravindar Thursday, January 22, 2015

Friend, Have you got any treatment for your daughter, Please help, Even my sons also facing the same issue, please share opinion on stem cells. Your information makes me to go right direction.

khalid-23 Monday, February 27, 2012

I am 51 years old and suffering from muscular dystrophy from my childhood.I can not stand with my own support and cant not claim stairs without support .More can not walk more than half km.My weight is 90 kg.Pl tell any treatment.

jayavelu Friday, September 2, 2011

mY SON IS UFFEREING THE MUSCULAR DYSTROPHY FOR THE LAST 4 YEARS KINLDY TELL US ANY MEIDCINE FOR THIS . ANY MEDICINE FOUND PLS LET US KNOW THIS E MAIL ID. jayavelu@gemsl.com

obaid Saturday, February 28, 2009

I am 43 years old person with muscular destropy and now Im bed ridden so please inform me any good news or new research result come on this desease. m. obaid ur rehman khan aligarh u.p india

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