FSHD 'Xenograft' Mouse Model Developed

An MDA-supported team has developed mice with transplanted muscle tissue from people with facioscapulohumeral MD that is likely to speed research  

Charles Emerson, an MDA grantee at the University of Massachusetts Medical School, helped develop the new FSHD xenograft mouse model.
by Margaret Wahl on February 14, 2014 - 1:44pm

Researchers funded in part by MDA have created a new research mouse (mouse "model") that they say is likely to speed investigations in facioscapulohumeral muscular dystrophy (FSHD) and therapy development for this disease. It's expected to complement an existing FSHD mouse model.

The new FSHD mouse utilizes a xenograft strategy, a technology involving transplanting tissue from one species to another. In this case, small pieces of muscle taken from FSHD patients were successfully transplanted to the leg muscles of mice. The researchers say the muscle xenograft strategy used for the FSHD mouse model has potential utility in the study of other muscle diseases and studies of muscle aging.

MDA supported Fedik Rahimov at Harvard Medical School and Boston Children's Hospital and Charles Emerson at the University of Massachusetts Medical School in Worcester on this study, results of which were published online Jan. 22, 2014, in Human Molecular Genetics. Study team leader Kathryn Wagner has MDA support at Kennedy Krieger Institute in Baltimore for research on muscle regeneration and muscle scarring.

The investigators say the xenograft FSHD model will allow the study of human FSHD-affected muscle over time, including its response to experimental therapies, "with a degree of access and invasive experimental scrutiny that is not available in human subjects." They say the new model's strength will be in providing researchers with molecular information about FSHD-affected muscle tissue, but it won't provide whole-body outcomes, such as grip strength or treadmill endurance.

FSHD field also has D4Z4-2.5 mouse

Last year, a different group of MDA-supported researchers developed the D4Z4-2.5 mouse model of FSHD, which mimics aspects of type 1 and type 2 FSHD, both of which result from abnormal production of a protein called DUX4. The D4Z4-2.5 mouse model produces the DUX4 protein in some muscle cells and showed more scarring and less muscle regeneration compared to normal mice a month after muscle injury.

The two models are expected to provide different kinds of information.

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