MDA SCIENTISTS SHOW THAT BOOSTING PROTEIN
COULD TREAT MUSCULAR DYSTROPHIES

TUCSON, Ariz., March 19, 2001 -- Researchers at the University of Illinois at Champaign-Urbana have performed experiments in mice that may lead to a new approach for treating several types of muscular dystrophy, the Muscular Dystrophy Association (MDA) announced today.

In today's issue of the Journal of Cell Biology, MDA-funded researchers Dean Burkin and Stephen Kaufman of the university's Department of Cell and Structural Biology and others, show that increasing the amount of alpha-7-beta-1 integrin protein in muscle cells can prevent muscular dystrophy in mice that otherwise would develop it.

In mice genetically engineered to lack the muscle proteins utrophin and dystrophin, the addition of genes that led to extra production of integrin resulted in relatively healthy mice instead of mice with muscular dystrophy. Untreated mice missing utrophin and dystrophin develop a severe muscle disease resembling human Duchenne muscular dystrophy.

All three proteins play key roles in maintaining the structural and functional integrity of muscle fibers.

"The finding that extra integrin can compensate for the loss of key muscle proteins adds an important new strategy to our growing arsenal in the fight against the muscular dystrophies," said Sharon Hesterlee, MDA director of Research Development. "We're vigorously pursuing this potential therapy, along with many others."

All nine forms of muscular dystrophy result from mutations in genes for specific muscle proteins. MDA researchers around the world are working on strategies for inserting functional genes to compensate for those specifically flawed in each dystrophy.

Besides gene insertion, another possible strategy would be to give patients a drug to increase their output of alpha-7-beta-1 integrin.

"If we could promote production of more integrin in a patient, that might be quite helpful" in making up for other missing or flawed proteins, Kaufman said.

MDA is a voluntary health agency working to defeat neuromuscular diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education. The Association's programs are funded almost entirely by individual private contributors.