Study Of Novel Approach To Treating Muscular Dystrophy At Boston Biomedical Research Institute Receives Noted Grant Award

WATERTOWN, Mass., Jan. 4 /PRNewswire/ -- An innovative research study at Boston Biomedical Research Institute targeting specialized stem cells in adult muscle known as “satellite cells,” has received a funding award from the Muscular Dystrophy Association. This novel approach by Dr. Xingbin Ai focuses on molecules known as heparan sulfatases, which are critical to maintaining the number of muscle stem cells in normal muscle and regulating stem cell activity in injured or diseased muscle.

Satellite cells are essential for muscle regeneration after injury induced by physiological stress or disease. Although normally a very small group of dormant cells in muscle, satellite cells have extensive capacities to expand themselves in response to muscle injury. During the course of muscle regeneration, a large majority of satellite cells transform to generate new muscle, while a subset of satellite cells remain in the dormant pool of satellite cells in the muscle. The number and regenerative capacities of satellite cells decrease with aging, and decrease much more significantly in patients with muscle dystrophic diseases. Consequently, muscle damage is poorly repaired by the elderly and by muscular dystrophy patients, leading to restricted movement and often, death.

“The therapeutic application of stem cells offers much hope to people suffering from muscular dystrophy and compromised mobility due to aging,” says Dr. Ai, “but this approach is challenged by the technical difficulties of obtaining large numbers of stem cells and systematically delivering them to injured muscles.”

To combat this challenge, Dr. Ai and her colleagues in the laboratory of Dr. Charles Emerson are studying the series of ‘signals’ that control the function of satellite cells during muscle regeneration. Specifically, they are examining the process in which the signals are regulated by heparan sulfatases. By understanding how the sulfatases maintain the number of muscle stem cells in healthy muscle, and how they regulate stem cell activity to repair injured muscle, Dr. Ai and her group hope to provide a basis for the use of heparan sulfate or heparan sulfate derivatives in muscle stem cell engineering for the treatment of Muscular Dystrophy as well as muscle weakness that accompanies normal aging.

This novel study at Boston Biomedical was recognized by a prestigious grant from the Muscular Dystrophy Association, a well-known national funder which combats neuromuscular diseases through worldwide research, medical and community services and public health education.

“We are delighted to have this very promising step forward in the fight against muscular dystrophy and aging-related muscle weakness recognized by such a notable funder of biomedical research,” said Boston Biomedical’s Director, Dr. Charles Emerson. “We are confident that these studies will help build a novel approach to treating this degenerative disease.”

Boston Biomedical Research Institute is a not-for-profit institution dedicated to the understanding, treatment and prevention of specific human diseases including muscular dystrophy, cancer, cardiovascular disease, Alzheimer’s disease, diabetes and conditions such as obesity and reproductive health problems. For more information visit http://www.bbri.org.

Media Contact: Terence McGowan, 617-658-7707, mcgowan@bbri.org

Boston Biomedical Research Institute

CONTACT: Terence McGowan of Boston Biomedical Research Institute,+1-617-658-7707, mcgowan@bbri.org

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