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 Custom-tailored strategy against glioblastomas
26.09.2016 | Rheinische Friedrich-Wilhelms-Universität Bonn

nachricht New leukemia treatment offers hope
23.09.2016 | King Abdullah University of Science and Technology

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: New welding process joins dissimilar sheets better

Friction stir welding is a still-young and thus often unfamiliar pressure welding process for joining flat components and semi-finished components made of light metals.
Scientists at the University of Stuttgart have now developed two new process variants that will considerably expand the areas of application for friction stir welding.
Technologie-Lizenz-Büro (TLB) GmbH supports the University of Stuttgart in patenting and marketing its innovations.

Friction stir welding is a still-young and thus often unfamiliar pressure welding process for joining flat components and semi-finished components made of...

Im Focus: First quantum photonic circuit with electrically driven light source

Optical quantum computers can revolutionize computer technology. A team of researchers led by scientists from Münster University and KIT now succeeded in putting a quantum optical experimental set-up onto a chip. In doing so, they have met one of the requirements for making it possible to use photonic circuits for optical quantum computers.

Optical quantum computers are what people are pinning their hopes on for tomorrow’s computer technology – whether for tap-proof data encryption, ultrafast...

Im Focus: OLED microdisplays in data glasses for improved human-machine interaction

The Fraunhofer Institute for Organic Electronics, Electron Beam and Plasma Technology FEP has been developing various applications for OLED microdisplays based on organic semiconductors. By integrating the capabilities of an image sensor directly into the microdisplay, eye movements can be recorded by the smart glasses and utilized for guidance and control functions, as one example. The new design will be debuted at Augmented World Expo Europe (AWE) in Berlin at Booth B25, October 18th – 19th.

“Augmented-reality” and “wearables” have become terms we encounter almost daily. Both can make daily life a little simpler and provide valuable assistance for...

Im Focus: Artificial Intelligence Helps in the Discovery of New Materials

With the help of artificial intelligence, chemists from the University of Basel in Switzerland have computed the characteristics of about two million crystals made up of four chemical elements. The researchers were able to identify 90 previously unknown thermodynamically stable crystals that can be regarded as new materials. They report on their findings in the scientific journal Physical Review Letters.

Elpasolite is a glassy, transparent, shiny and soft mineral with a cubic crystal structure. First discovered in El Paso County (Colorado, USA), it can also be...

Im Focus: Complex hardmetal tools out of the 3D printer

For the first time, Fraunhofer IKTS shows additively manufactured hardmetal tools at WorldPM 2016 in Hamburg. Mechanical, chemical as well as a high heat resistance and extreme hardness are required from tools that are used in mechanical and automotive engineering or in plastics and building materials industry. Researchers at the Fraunhofer Institute for Ceramic Technologies and Systems IKTS in Dresden managed the production of complex hardmetal tools via 3D printing in a quality that are in no way inferior to conventionally produced high-performance tools.

Fraunhofer IKTS counts decades of proven expertise in the development of hardmetals. To date, reliable cutting, drilling, pressing and stamping tools made of...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

HLF: From an experiment to an establishment

29.09.2016 | Event News

European Health Forum Gastein 2016 kicks off today

28.09.2016 | Event News

Laser use for neurosurgery and biofabrication - LaserForum 2016 focuses on medical technology

27.09.2016 | Event News

 
Latest News

New Multiferroic Materials from Building Blocks

29.09.2016 | Materials Sciences

Silicon Fluorescent Material Developed Enabling Observations under a Bright “Biological Optical Window”

29.09.2016 | Materials Sciences

X-shape Bio-inspired Structures

29.09.2016 | Interdisciplinary Research

VideoLinks
B2B-VideoLinks
More VideoLinks >>>