Stem cell therapy counters muscular dystrophy in mice: study

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WASHINGTON (AFP) - Scientists have demonstrated that a combination of

stem cell and gene therapy can be used to correct the devastating

hereditary disease of muscular dystrophy, according to a new study

published Wednesday.

Working with mice but using human cells, a team led by Yvan Torrente

of the University of Milan, Italy showed that they could spark the

most common form of the muscle-wasting disease, Duchenne muscular

dystrophy (DMD), and then reverse it, offering hope to millions of

sufferers world-wide.

They also showed that a DMD sufferer's own cells might be used for the

therapy, reducing the possibility of rejection.

DMD is a hereditary affliction in which the gene which helps

synthesize the key muscle protein dystrophin is mutated. Dystrophin is

essential to preventing damage to the muscles as they are used, and so

with the presence of the defective gene, its production is disturbed

and the muscles deteriorate.

In their experiment, Torrente and his team obtained muscle stem cells,

labelled CD133+, from human DMD patients. The cells were implanted

into mice where they generated defective muscle tissue resembling that

in the human DMD patients.

The researchers then took more muscle stem cells from the humans and

repaired the damaged dystrophin gene in them, so that dystrophin

production would be normalized.

Those cells were implanted in the same mice, and began producing

healthy muscle tissue, helping the mice begin to recover from the

induced DMD.

" These data demonstrate that genetically engineered blood or

muscle-derived CD133+ cells represent a possible tool for future stem

cell-based autograft applications in humans with DMD, " Torrente said

in a summary of the research.

" Additional research will substantially enhance our understanding of

the mechanisms underlying this effect, and may lead to the improvement

of gene and cell therapy strategies for DMD, " he added.

The research is published in the December issue of the journal Cell

Stem Cell.