The Italian Almanac

stem cells

Italian Science - January 18

Italian scientists report a possible solution to a major problem plaguing researchers working on gene therapy for muscular dystrophy: how to get the right gene to the right place to stop the muscle-wasting process that cripples people with the condition. A newly discovered kind of stem cell has successfully carried a corrective gene to muscles in mice with one form of muscular dystrophy, says a report in the July 11 issue of Science . The researchers are based at the Stem Cell Research Institute in Milan and the Institute of Cell Biology and Tissue Engineering in Rome.

It's much too early to think about human trials, says Sharon Hesterlee, director of research and development at the Muscular Dystrophy Association, but "this is a really significant piece of work. It's the first time we've been able to do a stem cell transplant and see an actual increase in function." The researchers used a kind of cell they discovered only last year and have named mesoangioblasts. These cells are found in blood vessel walls and are stem cells, meaning they can transform themselves into a variety of other cell types -- blood, bone, muscle and connective tissue. Most important, the Italians have found, they can migrate into muscle cells around blood vessels, carrying genes with them.

In the newly reported experiments, the gene for alpha sarcoglycan, a protein whose mutation or absence causes a form of muscular dystrophy, was inserted into mesoangioblasts, which were then grown in cell cultures. The engineered cells were injected into the arteries of mice lacking the gene. Three months later, the researchers found active alpha sarcoglycan proteins in the muscles of the treated mice, downstream from the injection site. The treated muscles contained a large number of apparently normal fibers, and the mice were able to walk on a rotating wheel longer than untreated animals, although not as long as healthy mice.