Muscle stem cells show promise against muscular dystrophy in mouse model

July 03, 2002

Scientists have isolated special muscle-generating stem cells that can improve muscle regeneration and deliver the missing protein dystrophin to damaged muscles in a mouse muscular dystrophy (MD) model, according to a study supported in part by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).

The research, carried out by Johnny Huard, Ph.D., and his colleagues at Children's Hospital of Pittsburgh and the University of Bonn in Germany, involves the isolation of a subpopulation of muscle stem cells with special characteristics: 1) they grow and reproduce over many generations; 2) they maintain their essential character through multiple generations; 3) they can differentiate into other kinds of cells needed to regenerate muscle tissue; and 4) they resist rejection by a foreign immune system. When these cells were transplanted into the mdx mouse, an animal model of muscular dystrophy, numerous dystrophin muscle fibers developed, suggesting that transplanting these cells into dystrophic muscle could potentially help restore function for a time.

The results may also signal that some of the major obstacles to muscle cell transplantation -- low cell survival rate, poor spreading of cells, and cell rejection by the immune system -- are being overcome. This, say the scientists, could make treatments for MD and other muscle-related diseases more effective.
The Muscular Dystrophy Association, the Parent Project Muscular Dystrophy Research and the Children's Hospital of Pittsburgh also helped fund the research project.

Muscular dystrophy refers to a group of genetic diseases characterized by progressive weakness and degeneration of the skeletal or voluntary muscles which control movement. The muscles of the heart and some other involuntary muscles are also affected in some forms of MD, and a few forms involve other organs, as well. There are currently no specific treatments for any of the forms of MD.

The mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), a part of the federal National Institutes of Health, 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 NIAMS, call our information clearinghouse at 1-877-22-NIAMS or visit the NIAMS Web site at

Qu-Petersen Z, Deasy B, Jankowski R, Ikezawa M, Cummins J, Pruchnic R, Mytinger J, Cao B, Gates C, Wernig A, Huard J. Identification of a novel population of muscle stem cells in mice: potential for muscle regeneration. J Cell Biology 2002;157(5):851-864.

To contact Dr. Huard, call Melanie Finnigan at Children's Hospital of Pittsburgh at (412) 692-5016.

For more information on muscular dystrophy, contact the following organizations:

Muscular Dystrophy Association
3300 East Sunrise Drive
Tucson, AZ 85718-3208
Tel: (520) 529-2000 or (800) 572-1717
Fax: (520) 529-5300

Muscular Dystrophy Family Foundation
2330 North Meridian Street
Indianapolis, IN 46208
Tel: (317) 923-6333 or (800) 544-1213
Fax: (317) 923-6334

Parent Project Muscular Dystrophy Research
1012 N. University Ave.
Middletown, OH 45044
Tel: (513) 424-0696 or (800) 714-KIDS
Fax: (513) 425-9907

Facioscapulohumeral (FSH) Society
3 Westwood Road
Lexington, MA 02420
Tel: (781) 860-0501
Fax: (781) 860-0599

International Myotonic Dystrophy Organization
P.O. Box 1121
Sunland, CA 91041-1121
Tel: (866) 679-7954 (toll free)

NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases

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