Scientist clears hurdles for muscular dystrop...(py mutated genes are replaced with healt...)

Date:10/29/2008

py, mutated genes are replaced with healthy genes. However, even with gene therapy, the healthy genes must reach every muscle in the body. Previously, scientists, including Duan's team, have experimented using viruses to deliver the healthy genes. However, these earlier studies were conducted in mice. Duan's team has now proven that this delivery system will reach every muscle in larger animals, such as dogs.

"Between 40 percent and 60 percent of the body weight is muscle, so it's vital that we find a way to deliver the therapy to every muscle in the body," Duan said. "Since dogs are 250 times the size of mice, but only nine times smaller than a human on average, we have taken a significant step in understanding if this therapy can work."

Duan's team has not stopped with just that discovery. In gene therapy, it is not feasible to fix every cell in the heart. Previously, scientists were uncertain whether partial correction could benefit patients. In an earlier study, Duan's research team demonstrated that heart tissue could be corrected enough to sustain a healthy life if only 50 percent of the tissue was affected by the therapy. Following the success with heart tissue, Duan's team has demonstrated for the first time that this result also is true with live heart muscle.

The Mizzou researchers delivered the therapy to the hearts of newborn mice with muscular dystrophy and found that gene therapy corrected many of the electrocardiogram abnormalities in these mice.

New tests have been developed to screen newborns with a high risk of muscular dystrophy. With few treatments available, the screening has not been widely accepted, but that may change if Duan's therapy proves to be effective.

"If you can treat an infant before they develop symptoms, you can treat the patient before they experience muscle loss," Duan said. "If you wait until symptoms start to appear, the muscle has already started to deteriorate. It's very diffic
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Contact: Christian Basi BasiC@missouri.edu 573-882-4430 University of Missouri-Columbia Source:Eurekalert

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