Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

New prospects for treating muscular dystrophy: Stem cells restore muscle in MD mice

11.07.2003


A study on mice suggests that a type of stem cells found in blood vessels may someday be able to regenerate wasting muscle in muscular dystrophy (MD) patients.



The authors caution that more research must be done before researchers consider applying these findings to humans. Nonetheless, their results provide a possible new direction for efforts that have met largely with frustration thus far. The study appears in the journal Science, published by AAAS, the science society.

The research team, led by Giulio Cossu of the Stem Cell Research Institute, in Milan, and the University of Rome and the Institute of Cell Biology and Tissue Engineering, in Rome, has found that these stem cells can cross from the bloodstream, into muscle tissue. There, they seem to take on a new identity, helping to generate new muscle fibers in mice with MD-like symptoms.


MD is a collection of disorders caused by genetic defects that lead to increasing muscle weakness over time. These disorders currently have no cure.

"Although these results are exciting, we have not cured the mice," Cossu said. "We believe this is a significant step toward therapy, but the question that keeps me awake at night is whether this will work in larger animals."

Cossu’s team conducted its experiments on mice with the same genetic defect that causes one form of MD in humans. If the same stem cells, called "mesoangioblasts," can be collected from human MD patients, and if the cells have the same versatility they do early in life, they may offer a new avenue for treating the disease.

The approach Cossu and his colleagues are envisioning would involve collecting mesoangioblasts from a patient’s blood vessels, genetically "correcting" the cells in the laboratory, allowing them to multiply, and then injecting the cells back into the patient’s bloodstream. The cells would then migrate to the patient’s muscles, and begin producing healthy muscle cells.

Because the cells would be from the patient’s own body, his or her immune system wouldn’t reject them.

Trying to find a therapy for MD "has been a long and frustrating series of exploits," said Cossu.

"There is this problem of delivering the cells, or in the case of gene therapy, the viral vector, to all of the muscles. If you could go through the circulatory system, you would have a way to homogeneously deliver the cells or the vector to all the muscle fibers," he said.

Several key issues must be answered before such a therapy can be developed for humans, according to the Science authors.

First, these particular stem cells are fairly new to scientists. Cossu and his colleagues discovered them approximately a year ago, and are still learning how to identify them and how they function in the body. Thus far, Cossu’s team has only isolated human mesoangioblasts from fetal blood vessels.

More research is also needed for the "genetic correction" step of the therapy, which involves inserting the healthy version of a gene into the stem cell. The lentivirus Cossu used for delivering the gene in his mouse study provided the efficiency the researchers needed, but poses serious safety concerns for humans. Whether the safer retrovirus would be up to the task must still be determined.

When Cossu and his colleagues first identified mesoangioblasts last year, they determined that these cells could differentiate into a variety of cell types, including blood, bone, muscle, and connective tissue. They also found that the cells migrated outside the blood vessel, in response to inflammation.

For the current study, the researchers injected mesoangioblasts into the arteries of mice lacking the alpha sarcoglycan gene. This gene is one of several that, when defective, cause a type of MD called limb-girdle muscular dystrophy.

The researchers detected a significant portion of the normal mesoangioblasts in the muscles downstream of the injected artery. They also experimented with genetically modified mesoangioblasts, restoring healthy versions of alpha sarcoglycan gene to the cells. Three months after a single injection, they found healthy alpha sarcoglycan proteins in the muscles of the treated mice.

When Cossu’s group examined the mice, they found that the treated muscles contained larger and more numerous and apparently normal muscle fibers. The treated animals were also able to walk on a rotating wheel for longer than untreated animals, although not as long as healthy mice.

"I’m convinced this is an important result, but this is still not the therapy -- for the mice or for patients," Cossu said.


Giulio Cossu’s co-authors are Maurilio Sampaolesi, Anna Innocenzi, Rossana Tonlorenzi, and M. Gabriella Cusella De Angelis of the Stem Cell Research Institute in Milan, Italy; M.G.C. De Angelis is also at the University of Pavia, in Pavia, Italy; Yvan Torrente and Nereo Bresolin of Ospedale Maggiore Policlinico, in Milan, Italy; N. Bresolin is also at Istituto E. Medea, Bosisio Parini, in Lecco, Italy; M. Antonietta Pellegrino and Roberto Bottinelli at University of Pavia, in Pavia Italy; and Rita Barresi and Kevin P. Campbell at Howard Hughes Medical Institute and the University of Iowa, in Iowa City, IA. The study was supported by Telethon/Fondazione Zegna, the European Community, Duchenne Parent Project Italia/Compagnia di San Paolo, Muscular Dystrophy Association, Fondazione Istituto Pasteur-Cenci Bolognetti, Associazione Italiana Ricera sul Cancro (AIRC), Agenzia Spaziale Italiana (ASI) and the Italian Ministry of Health.

Ginger Pinholster | EurekAlert!
Further information:
http://www.aaas.org/

More articles from Health and Medicine:

nachricht Correct connections are crucial
26.06.2017 | Charité - Universitätsmedizin Berlin

nachricht One gene closer to regenerative therapy for muscular disorders
01.06.2017 | Cincinnati Children's Hospital Medical Center

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Can we see monkeys from space? Emerging technologies to map biodiversity

An international team of scientists has proposed a new multi-disciplinary approach in which an array of new technologies will allow us to map biodiversity and the risks that wildlife is facing at the scale of whole landscapes. The findings are published in Nature Ecology and Evolution. This international research is led by the Kunming Institute of Zoology from China, University of East Anglia, University of Leicester and the Leibniz Institute for Zoo and Wildlife Research.

Using a combination of satellite and ground data, the team proposes that it is now possible to map biodiversity with an accuracy that has not been previously...

Im Focus: Climate satellite: Tracking methane with robust laser technology

Heatwaves in the Arctic, longer periods of vegetation in Europe, severe floods in West Africa – starting in 2021, scientists want to explore the emissions of the greenhouse gas methane with the German-French satellite MERLIN. This is made possible by a new robust laser system of the Fraunhofer Institute for Laser Technology ILT in Aachen, which achieves unprecedented measurement accuracy.

Methane is primarily the result of the decomposition of organic matter. The gas has a 25 times greater warming potential than carbon dioxide, but is not as...

Im Focus: How protons move through a fuel cell

Hydrogen is regarded as the energy source of the future: It is produced with solar power and can be used to generate heat and electricity in fuel cells. Empa researchers have now succeeded in decoding the movement of hydrogen ions in crystals – a key step towards more efficient energy conversion in the hydrogen industry of tomorrow.

As charge carriers, electrons and ions play the leading role in electrochemical energy storage devices and converters such as batteries and fuel cells. Proton...

Im Focus: A unique data centre for cosmological simulations

Scientists from the Excellence Cluster Universe at the Ludwig-Maximilians-Universität Munich have establised "Cosmowebportal", a unique data centre for cosmological simulations located at the Leibniz Supercomputing Centre (LRZ) of the Bavarian Academy of Sciences. The complete results of a series of large hydrodynamical cosmological simulations are available, with data volumes typically exceeding several hundred terabytes. Scientists worldwide can interactively explore these complex simulations via a web interface and directly access the results.

With current telescopes, scientists can observe our Universe’s galaxies and galaxy clusters and their distribution along an invisible cosmic web. From the...

Im Focus: Scientists develop molecular thermometer for contactless measurement using infrared light

Temperature measurements possible even on the smallest scale / Molecular ruby for use in material sciences, biology, and medicine

Chemists at Johannes Gutenberg University Mainz (JGU) in cooperation with researchers of the German Federal Institute for Materials Research and Testing (BAM)...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Plants are networkers

19.06.2017 | Event News

Digital Survival Training for Executives

13.06.2017 | Event News

Global Learning Council Summit 2017

13.06.2017 | Event News

 
Latest News

Touch Displays WAY-AX and WAY-DX by WayCon

27.06.2017 | Power and Electrical Engineering

Drones that drive

27.06.2017 | Information Technology

Ultra-compact phase modulators based on graphene plasmons

27.06.2017 | Physics and Astronomy

VideoLinks
B2B-VideoLinks
More VideoLinks >>>