News Releases

News Releases

For Immediate Release

Children’s Hospital of Pittsburgh Researcher Receives More than $1 million in NIH Funding to Seek Cure for Muscular Dystrophy

Children’s Hospital of Pittsburgh researcher Johnny Huard, PhD, has received a grant of almost $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.

Dr. Huard’s five-year grant will be used to research whether a unique population of stem cells he identified in mice last year can be transplanted into mice with a disease similar to Duchenne muscular dystrophy to promote muscle regeneration.

“We are finding that these unique muscle-derived stem cells we transplant into mice with muscular dystrophy do not die and are not rejected by the immune system – which had always happened in the past,” Dr. Huard said. “This grant from the NIH will allow us to study this further and may someday lead to a treatment for children with the disease.”

Duchenne muscular dystrophy is a genetic disease that causes muscles to weaken and can lead to heart problems, scoliosis and eventual death. It is the most common genetic illness in children and is estimated to affect one in every 3,500 boys.

Last May, Dr. Huard, director of the Growth and Development Laboratory at Children’s Hospital, and colleagues from the University of Pittsburgh published research identifying the unique stem cells. These stem cells are able to deliver a key protein, called dystrophin, to diseased muscles. This leads to improved muscle function, according to Dr. Huard.

Patients with Duchenne muscular dystrophy lack dystrophin, the protein that provides structure for cell membranes to adhere to cell material, which keeps the muscle intact.

Dr. Huard’s grant was one of 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.

“Research on stem cells is important in the search for prevention and cure of debilitating musculoskeletal diseases,” said Stephen I. Katz, MD, PhD, director of the NIAMS. “These studies will have a solid impact on the variety of diseases that may be treated in the future.”

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

Last Update
February 20, 2008
  • Increase/Decrease Text Size
  • Print This Page
Last Update
February 20, 2008