(Philadelphia, PA) – Researchers at the University of
Pennsylvania School of Medicine report a novel strategy for
stimulating the production of utrophin – an important muscle
protein in young mice – for muscular dystrophy therapy. The
investigators gave mdx mice (the mouse model for Duchenne's
muscular dystrophy) heregulin, a small molecule to turn on the
production of utrophin in their muscles. Utrophin improved
muscle function in the mdx mice. "Our strategy boosts the levels
of an existing gene using pre-existing cellular machinery rather
than having to deliver a gene via gene therapy," says lead
author Tejvir S. Khurana, MD, PhD, Assistant Professor of
Physiology & Member of the Pennsylvania Muscle Institute. They
detected an approximately threefold increase of utrophin levels
over control mdx mice. "This is the level at which one starts
seeing a therapeutic affect, as measured in lab tests with mouse
muscles," says Khurana. The researchers noted an improvement in
the quality of mouse muscle tissue, the bi