(Press-News.org) Stem cells from patients with a rare form of muscular dystrophy have been successfully transplanted into mice affected by the same form of dystrophy, according to a new study published today in Science Translational Medicine.
For the first time, scientists have turned muscular dystrophy patients' fibroblast cells (common cells found in connective tissue) into stem cells and then differentiated them into muscle precursor cells. The muscle cells were then genetically modified and transplanted into mice.
The new technique, which was initially developed at the San Raffaele Scientific Institute of Milan and completed at UCL, could be used in the future for treating patients with limb-girdle muscular dystrophy (a rare form in which the shoulders and hips are primarily affected) and, possibly, other forms of muscular dystrophies.
Muscular dystrophies are genetic disorders primarily affecting skeletal muscle that result in greatly impaired mobility and, in severe cases, respiratory and cardiac dysfunction. There is no effective treatment, although several new approaches are entering clinical testing including cell therapy.
In this study, scientists focused on genetically modifying a type of cell called a mesoangioblast, which is derived from blood vessels and has been shown in previous studies to have potential in treating muscular dystrophy. However, the authors found that they could not get a sufficient number of mesoangioblasts from patients with limb-girdle muscular dystrophy because the muscles of the patients were depleted of these cells.
Instead, scientists in this study "reprogrammed" adult cells from patients with limb-girdle muscular dystrophy into stem cells and were able to induce them to differentiate into mesoangioblast-like cells. After these 'progenitor' cells were genetically corrected using a viral vector, they were injected into mice with muscular dystrophy, where they homed-in on damaged muscle fibres.
The researchers also showed that when the same muscle progenitor cells were derived from mice the transplanted cells strengthened damaged muscle and enabled the dystrophic mice to run for longer on a treadmill than dystrophic mice that did not receive the cells.
Dr Francesco Saverio Tedesco, UCL Cell & Developmental Biology, who led the study, said: "This is a major proof of concept study. We have shown that we can bypass the limited amount of patients' muscle stem cells using induced pluripotent stem cells and then produce unlimited numbers of genetically corrected progenitor cells.
"This technique may be useful in the future for treating limb-girdle muscular dystrophy and perhaps other forms of muscular dystrophy."
Professor Giulio Cossu, another UCL author, said: "This procedure is very promising, but it will need to be strenuously validated before it can be translated into a clinical setting, also considering that clinical safety for these "reprogrammed" stem cells has not yet been demonstrated for any disease."
INFORMATION:
Notes for Editors
1. For more information or to interview the UCL authors, please contact Clare Ryan in the UCL Media Relations Office on tel: +44 (0)20 3108 3846, mobile: +44 07747 565 056, out of hours +44 (0)7917 271 364, e-mail: clare.ryan@ucl.ac.uk.
2. "Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy." is published online today in Science Translational Medicine. Journalists can obtain copies of the paper by contacting UCL Media Relations.
3. Images from the study are available on request from UCL Media Relations.
4. This work was supported by European Community 7th Framework project OPTISTEM, Duchenne Parent Project Italy, Telethon Network of Genetic Biobanks and the European Research Council.
About UCL (University College London)
Founded in 1826, UCL was the first English university established after Oxford and Cambridge, the first to admit students regardless of race, class, religion or gender, and the first to provide systematic teaching of law, architecture and medicine. We are among the world's top universities, as reflected by performance in a range of international rankings and tables. UCL currently has 24,000 students from almost 140 countries, and more than 9,500 employees. Our annual income is over £800 million.
www.ucl.ac.uk | Follow us on Twitter @uclnews
END
Medical residents are an essential part of the hospital workforce. Although still in training the take on much of the day to day care of patients. A systematic review published in BioMed Central's open access journal BMC Medicine shows that patient by properly supervised residents care is safe and of equal quality to that of fully trained doctors.
Residency training is an essential part of a doctors education after they leave university. Once completed doctors are expected to provide high quality care and while many studies have looked at different aspects of residency ...
Standing for long periods during pregnancy may curb the growth of the developing fetus, suggests research published online in Occupational and Environmental Medicine.
Previous research has indicated that long working hours may increase the risk of birth defects, premature birth, stillbirth and low birthweight.
The researchers assessed the fetal growth rates of 4680 mums to be from early pregnancy onwards between 2002 and 2006.
Midway through their pregnancy, the women were quizzed about their work conditions and the physical demands of their jobs, including whether ...
A diet rich in vegetables could help stave off the development of the serious condition acute pancreatitis, suggests a large study published online in the journal Gut.
Pancreatitis refers to inflammation of the pancreas - the gland behind the stomach, which, among other things, releases digestive enzymes to break down food.
Occasionally these enzymes become active inside the pancreas, and start to digest the gland itself. In up to one in five of those with acute pancreatitis symptoms are severe and potentially life threatening.
Previous research suggests that excessive ...
A heart attack doesn't just damage heart muscle tissue by cutting off its blood supply, it also sets off an inflammatory cascade that worsens underlying atherosclerosis, actively increasing the risk for a future heart attack. These findings from a study receiving advance online publication in Nature suggest an important new therapeutic strategy for preventing heart attacks and strokes, both of which are caused when atherosclerotic plaques rupture and block important blood vessels.
"We have known for a long time that heart attack patients are at increased risk for ...
Geckos are known for sticky toes that allow them to climb up walls and even hang upside down on ceilings. A new study shows that geckos have gained and lost these unique adhesive structures multiple times over the course of their long evolutionary history in response to habitat changes.
"Scientists have long thought that adhesive toepads originated just once in geckos, twice at the most," says University of Minnesota postdoctoral researcher Tony Gamble, a coauthor of the study. "To discover that geckos evolved sticky toepads again and again is amazing."
The findings ...
Move over Elle Macpherson – the search is now on for the pig with the best legs as part of a new research project to improve the health and welfare of pigs on farms across the UK.
The study, being led by Newcastle University, UK, was set up to see if we can predict from an early age whether a pig is at risk of becoming lame, simply by analysing the way it walks.
Using video motion capture – a technique similar to that used in animation for Hollywood blockbusters such as Avatar and Lord of the Rings – the team measured changes in the pigs' gait, focussing on the angle ...
Scientists have identified a new mechanism of bacterial pathogenesis. The results of the research project, partly funded by the Academy of Finland, have been published in the journal Proceedings of the National Academy of Sciences of the United States of America (PNAS).
Bacteria that cause chronic infections have an amazing but yet poorly known ability to subvert immune response, live and produce offspring, enter and wake up from a dormant phase to cause, in some instances, deadly complications.
Bartonella bacteria cause chronic infections in mammals (incl. humans), ...
A new study confirms that doing short journeys on foot rather than taking the car or motorbike would avoid the death of 108 men and 79 women a year in Catalonia alone. This would imply annual savings of more than 200 million euros.
Researchers from the World Health Organisation (WHO) and the Barcelona Public Health Agency (ASPB), headed by Catherine Pérez from the Healthcare Information Systems Service, have estimated the yearly economic benefits from a reduction in death rates by substituting at least one short vehicle journey for a walk.
The authors conducted a ...
A condition that temporarily causes heart failure in people who experience severe stress might actually protect the heart from very high levels of adrenaline, according to a new study published in the journal Circulation. The research provides the first physiological explanation for Takotsubo cardiomyopathy, also called "broken heart syndrome" because it affects people who suffer severe emotional stress after bereavement, and suggests guidance for treatment.
Around 1-2% of people who are initially suspected of having a heart attack are finally discovered to have this ...
The exhaust from diesel-fuelled vehicles, wood fires and coal-driven power stations contains small particles of soot that flow out into the atmosphere. The soot is a scourge for the climate but also for human health. Now for the first time, researchers have studied in detail how diesel soot gets stuck in the lungs. The results show that more than half of all inhaled soot particles remain in the body.
The figure is higher than for most other types of particles. For example "only" 20 per cent of another type of particle from wood smoke and other biomass combustion gets ...
