RESEARCH NEWS
MORE ABOUT "SUPER-MICE"

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In a breakthrough that might lead to better treatment for muscular dystrophy, MDA-funded researchers have created superstrong mice that are largely invulnerable to age-related muscle frailty. The following Q&A addresses some important issues about the research.

Q:  How is aging relevant to muscular dystrophy?

A:  Like muscular dystrophy, aging is associated with a loss of muscle mass and a decreased capacity to recover from muscle injuries. Some researchers think that aging and muscular dystrophy might cause this muscle frailty in fundamentally similar ways.

Q:  How are the mice able to resist muscle frailty?

A:  The mice were genetically engineered to overproduce a protein called muscle insulinlike growth factor 1 (mIgf1, for short). The protein is normally produced by young, healthy muscles, and it stimulates muscle growth and regeneration. Boosting mIgf1 levels in the mice made them more muscular throughout life, and, in old age, it enabled them to recover from muscle injuries that would have normally caused permanent damage.

Q:  Can mIgf1 be used to treat muscular dystrophy?

A:  Maybe. So far, the results are highly promising, but more research is required before mIgf1 can be used to treat people. For example, the effects of mIgf1 still need to be tested in mice that have muscular dystrophy.

Q:  How would mIgf1 be used to treat muscular dystrophy in people?

A:  MIgf1 would probably be administered by gene therapy technology that's currently under development. Before the first human trials can take place, a suitable gene-delivery vehicle (vector) must be developed, and Food and Drug Administration approval must be secured.

Q:  If all goes well, when will mIgf1 treatment become available?

A:  The researchers who developed the mIgf1 mice estimate that clinical trials in muscular dystrophy could start in as little as two years, but more time could be required for testing and regulatory approval.

For more information about this topic, please read the press release,
MDA Researchers Create Super-Mice Resistant to Age-Related Muscle Wasting.