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Adult stem cells used to correct DMD in mice

17 December 2007
Appeared in BioNews 438

Adult stem cells harvested from human patients with the muscle wasting disease Duchene Muscular Dystrophy (DMD) can be genetically corrected and used to improve muscle strength in mice with DMD, according to a study published in the journal Cell Stem Cell last week. The researchers, based at the University of Milan, heralded the new gene therapy as a step towards being able to treat DMD patients with their own cells, thus avoiding the tissue rejection risks associated with foreign tissue transplants.

DMD is a muscle-wasting disease that causes a steady deterioration of muscles and often results in death before the age of 30. The condition, which is usually inherited and incurable, affects 1 in 3500 male newborn boys. The disease is caused by an inherited fault in the gene that encodes the body's instructions for making dystrophin - a crucial muscle protein.

The research team isolated so-called 'progenitor' muscle cells - a type of adult stem cell that can grow into functioning muscle cells - from human DMD patients and injected them into mice to show that the mice displayed DMD-like symptoms. They then rescued the mice by again injecting them with progenitor muscle cells from the same patients, which had been genetically modified to make them ignore the mutation, producing a shortened but functioning version of the dystrophin protein.

Within three weeks the researchers saw marked improvements in the mice, including increased dystrophin levels in muscle fibres and increased muscle strength, as demonstrated by comparing the mice running on a treadmill before and after treatment and by lab tests carried out on leg muscles removed from the mice. 'Use of the patient's own cells would reduce the risk of implant rejection seen with transplantation of normal muscle-forming cells', explains Dr Yvan Torrente, who led the study.

Although genetic modification and muscle transplant techniques used in this study have both been tried before, this is the first time they have been studied in unison, note Kay Davis, Oxford University, and Miranda Grounds, University of Western Australia, in an accompanying review article.

Muscular Dystrophy: Reprogrammed Human Adult Stem Cells Rescue Diseased Muscle In Mice
Science Daily |  14 December 2007
Stem cells for Duchenne?
The Scientist |  14 December 2007
Stem cell therapy counters muscular dystrophy in mice: study
AFP |  13 December 2007
9 March 2009 - by Rosie Beauchamp 
It was reported this week in the journal Stem Cell that a group of researchers from the University of New South Wales, Australia, have made a major breakthrough in the success of regrowing damaged muscle tissue using adult stem cells. Previous research carried out at the University...
27 January 2008 - by Dr Rebecca Robey 
Researchers at the University of Texas Southwestern Medical Centre have developed a new technique to treat the symptoms of muscular dystrophy using embryonic stem (ES) cells. The group, reporting in the February 2008 issue of Nature Medicine, successfully manipulated mouse ES cells to transform them into muscle-forming...
7 January 2008 - by Ailsa Stevens 
An experimental treatment for boys with the inherited muscle wasting disease Duchene Muscular Dystrophy (DMD) has showed promise in human safety trials, according to a study published in the New England Journal of Medicine. In the first ever trial on humans, the new drug was shown to...
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