Spotlight on Research 2011

September 2011 (historical)

Researchers Achieve Needed Levels of Stem Cells to Treat Muscle Disease in Mice

Transplanting stem cells into diseased muscle to help generate new muscle has been a somewhat successful strategy for scientists working on animal models of genetic muscle diseases like muscular dystrophy (MD). One problem, researchers say, has been cultivating large enough quantities of sufficiently mature cells to improve muscle function in animals—and, eventually, in people with muscle diseases. Recently, however, a University of Minnesota research team led by Rita Perlingeiro, Ph.D., and supported, in part, by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), used a new technique to produce enough cells to both ensure the transplantation and survival of the cells in mice, and to restore some lost muscle contractile properties. The new study, reported in Stem Cell Review and Report, is considered an important step in developing cell therapies for MD, traumatic muscle injury and age-related muscle loss.

The Minnesota team achieved their results by developing induced pluripotent stem (iPS) cells from healthy mice in cell culture. iPS cells have the potential to produce different kinds of specialized cells, and the scientists experimentally induced the expression of Pax7—a DNA-binding protein normally involved in the development and regeneration of muscle—to the developing iPS cells. As a result, the scientists were able to successfully produce muscle progenitor cells—precursors to muscle cells—at a rate much higher than those previously observed in other studies.

The researchers then injected the new progenitor cells into the muscles of mice with MD. Not only did they survive and incorporate well in the diseased muscle tissue, but the treated muscles were able to generate more force than control nontreated muscles in the same animal. "Our results point toward the potential success of iPS-based stem cell transplantation for human muscle disease,” says Dr. Perlingeiro. “There are a number of other issues that remain to be resolved, but overall, we consider it a significant step forward."

Partial support for the study was also provided by the National Heart, Lung, and Blood Institute, a component of the NIH, and the Dr. Bob and Jean Smith Foundation.

The mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), a part of the U.S. Department of Health and Human Services’ National Institutes of Health (NIH), is to support research into the causes, treatment, and prevention of arthritis and musculoskeletal and skin diseases; the training of basic and clinical scientists to carry out this research; and the dissemination of information on research progress in these diseases. For more information about the NIAMS, call the information clearinghouse at (301) 495-4484 or (877) 22-NIAMS (free call) or visit the NIAMS website at


Darabi R, Pan W, Bosnakovski D, Baik J, Kyba M, Perlingeiro RC. Functional Myogenic Engraftment from Mouse iPS Cells. Stem Cell Rev. 2011 Apr 2.