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A Long-Awaited Mouse Offers a New Animal Model for FSH Muscular Dystrophy
Date:4/4/2013

Lexington, MA (PRWEB) April 04, 2013

It’s a truism of medical research that humans are cheaper and better to study than animals. People don’t require sterile cages, gourmet chow or 24/7 monitoring by a battalion of technicians. And because of species differences, diseases and cures in animals often don’t accurately portray the human condition. Still, animals remain invaluable because they can be manipulated and probed in ways that human volunteers (and ethical boards) might frown upon.

When a disease is unique to humans, creating an animal model presents a particular challenge. Such has been the case with facioscapulohumeral muscular dystrophy (FSHD), one of the most common muscular dystrophies. Past efforts resulted in mice that exhibited features of FSHD, but which required cranking up the expression of genes under conditions that researchers fear were too dissimilar to the human disorder.

Now an international team has published a mouse model that appears more promising. The result of a decade’s worth of work, during which scientific understanding of FSHD exploded, the new mouse was published today in an exhaustive, 58-page article in PLoS Genetics. “We hope that in the near future these mouse models will serve an important purpose in drug development programs for FSHD,” remarked senior author Silvère van der Maarel of Leiden University in the Netherlands.

Other members of the team work at the Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; King’s College London, United Kingdom; Radboud University Nijmegen Medical Centre, Nijmegen, Netherlands; and the University of Rochester Medical Center, Rochester, New York, USA.

The herculean project was initiated in 2003 by the FSH Society’s Marjorie Bronfman Fellowship grant. The patient-driven charity was seeking a
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