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Collaborative Success Stories: Needle in a DNA-Stack


University of Missouri scientists have discovered genetic clues to a new muscular dystrophy treatment.

Children with Duchenne muscular dystrophy (DMD) face a future of rapidly weakening muscles, usually leading to death in early adulthood. Unfortunately, a cure has eluded scientists.

Normally, sequences in dystrophin cause the gene to interact with essential proteins, ensuring sufficient blood supply to muscles. In DMD, however, mutations in the gene sequence disrupt the interaction—reducing oxygen supply and causing muscle cell death. Remarkably, MU investigators Drs. Dongsheng Duan and Yi Lai have identified the key sequence that is responsible for the interaction.

The dystrophin gene is the largest gene found in nature, so identifying the sequence was a huge undertaking. Dr. Scott Harper, a muscular dystrophy expert at Ohio State University, said the MU researchers “found the proverbial needle in a haystack.”

Using their results, the scientists treated young dogs with early DMD symptoms. The animals were injected with a miniature version of the dystrophin gene and a common, non-symptomatic virus which spread the microgene through the body. The dogs continued to develop normally, even several months down the line. The National Institutes of Health awarded them $3 million to continue their promising research.

Dr. Dongsheng Duan is a professor of medical research and Dr. Yi Lai is an assistant research professor. Both scientists are housed in the department of molecular microbiology and immunology at MU’s School of Medicine.

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Related Initiative(s):
One Health/One Medicine