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For Immediate Release

Children’s Hospital of Pittsburgh Researchers Find Stem Cells With the Potential to Repair Muscle and Produce Blood Cells

New findings by Johnny Huard, PhD, and team to be published in leading scientific journal

Children’s Hospital of Pittsburgh researchers have discovered that a unique population of muscle-derived stem cells has the potential to repair damaged muscle in muscular dystrophy patients; produce blood needed for life-saving transfusions; and improve outcomes for transplant recipients.

Johnny Huard, PhD, director of the Growth and Development Laboratory at Children’s, and his colleagues at the University of Pittsburgh first identified this unique population of muscle-derived stem cells last year. Since then, Dr. Huard’s team, including Baohong Cao, MD, PhD, of the University of Pittsburgh, have found that these muscle-derived stem cells are able to grow into blood cells while retaining the ability to produce muscle. It is the first time this ability - known as plasticity - has been demonstrated.

Dr. Huard said his findings may lead to life-saving applications in the hospital setting.

“Our findings show that this unique population of stem cells offers unlimited potential,” said Dr. Huard, an associate professor of Orthopaedic Surgery, Molecular Genetics and Biochemistry, and Bioengineering at the University of Pittsburgh School of Medicine. “We could someday be using muscle-derived stem cells to cure muscular dystrophy, to produce blood for life-saving transfusions and to prevent rejection in people receiving organ transplants.”

The study will be published in the July issue of Nature Cell Biology.

In the study, Dr. Huard’s team injected a unique population of stem cells into the blood of mice with a muscle condition similar to Duchenne muscular dystrophy, a genetic disease that causes muscle to weaken and can lead to heart problems, scoliosis and eventually death. Until now, researchers only had success injecting these stem cells directly into muscle, not into blood. By injecting the stem cells into the blood, the researchers determined that a small percentage of these stem cells regenerated the muscles that were ravaged by dystrophy. In dystrophic patients, the muscles lack dystrophin, a protein that provides structure and mechanical integrity to muscle cells.

Children’s researchers also discovered that when they injected stem cells into the blood, the percentage of stem cells working to repair damaged muscle increased when the damage was greater.

A second important application of Dr. Huard’s research is in the area of blood transfusions. Children’s researchers have discovered that the newly identified population of stem cells can be coaxed to become blood cells. These findings could make it possible to take a muscle biopsy from a patient in need of a blood transfusion, isolate stem cells from the biopsy and use those stem cells to produce blood for the patient.

“For instance, if you were in need of an emergency blood transfusion and finding a match was difficult, we may be able to use your own stem cells taken from your muscle to produce the blood you need,” Dr. Huard said. “In the future, we might be able to take these biopsies at a young age, grow blood supplies for individuals and store the blood for use in emergencies when matches are not available.”

A third potential use for Dr. Huard’s research is with transplantation. Transplant recipients often suffer from rejection, a condition in which the recipient’s immune system attacks the new organ, causing it to fail.

Potentially, Dr. Huard said it might be possible to take muscle-derived stem cells from the donor prior to transplant and inject them into the intended recipient. These stem cells would then grow into blood cells that make up the immune system. Then, when the organ is transplanted, the recipient’s immune system will recognize the organ. With that, the chance for rejection decreases.

These findings are part of Dr. Huard’s ongoing investigation into the potential uses of the stem cell population he identified. In January of this year, Dr. Huard received a grant of nearly $1.2 million from the National Institutes of Health to study muscle regeneration in diseased mice - a treatment that eventually could lead to a cure for Duchenne muscular dystrophy (DMD). DMD is estimated to affect one in every 3,500 boys.

The grant was one of only five awarded to U.S. researchers in January by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). The NIAMS grants support research focusing on the use of adult stem cells to treat bone and muscle that may have been altered by disease.

Marc Lukasiak, 412-692-5016,
Melanie Finnigan, 412-692-5016,

Last Update
February 20, 2008
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Last Update
February 20, 2008