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Updated: Mar 19, 2026
Assessing Functional Performance in the Mdx Mouse Model
Published on: March 27, 2014
Treatment with rGDF11 does not improve the dystrophic muscle pathology of mdx mice
Fabrizio Rinaldi1, Yu Zhang1,2, Ricardo Mondragon-Gonzalez1,3
1Lillehei Heart Institute, Department of Medicine, University of Minnesota, 4-128 CCRB, 2231 6th St. SE, Minneapolis, MN 55455 USA.
Background:
Duchenne muscular dystrophy (DMD) is an inherited lethal muscle wasting disease characterized by cycles of degeneration and regeneration, with no effective therapy. Growth differentiation factor 11 (GDF11), a member of the TGF-β superfamily and myostatin homologous, has been reported to have the capacity to reverse age-related skeletal muscle loss. These initial findings led us to investigate the ability of GDF11 to promote regeneration in the context of muscular dystrophy and determine whether it could be a candidate to slow down or reverse the disease progression in DMD.
Results:
Here, we delivered recombinant GDF11 (rGDF11) to dystrophin-deficient mice using the intra-peritoneal route for 30 days and evaluated histology and function in both steady-state and cardiotoxin-injured muscles. Our data confirmed that treatment with rGDF11 resulted in elevated levels of this factor in the circulation. However, this had no effect on muscle contractility nor on muscle histology. Moreover, no difference was found in the number of regenerating myofibers displaying centrally located nuclei. On the other hand, we did observe increased collagen content, which denotes fibrosis, in the muscles of rGDF11-treated dystrophic mice.
Conclusions:
Taken together, our findings indicate no beneficial effect of treating dystrophic mice with rGDF11 and raise caution to a potential harmful effect, as shown by the pro-fibrotic outcome.
Insights
Growth differentiation factor 11 (GDF11) did not improve muscle regeneration in Duchenne muscular dystrophy (DMD) mice. Instead, GDF11 treatment led to increased fibrosis, suggesting potential harm.
Area of Science:
- Muscle regeneration and disease
- Biologics and therapeutic targets
Background:
- Duchenne muscular dystrophy (DMD) is a fatal genetic disorder causing progressive muscle wasting with no current effective treatments.
- Growth differentiation factor 11 (GDF11), a protein related to myostatin, has shown potential in reversing age-related muscle loss.
Purpose of the Study:
- To investigate the efficacy of GDF11 in promoting muscle regeneration in the context of muscular dystrophy.
- To determine if GDF11 could slow or reverse disease progression in Duchenne muscular dystrophy.
Main Methods:
- Recombinant GDF11 (rGDF11) was administered intraperitoneally to dystrophin-deficient mice for 30 days.
- Histology, muscle function, and markers of regeneration (centrally located nuclei) were evaluated in steady-state and injured muscles.
Main Results:
- rGDF11 treatment led to elevated circulating levels of GDF11 but did not improve muscle contractility or histology.
- No significant difference was observed in the number of regenerating myofibers with centrally located nuclei.
- Increased collagen content, indicative of fibrosis, was observed in the muscles of rGDF11-treated mice.
Conclusions:
- Treatment with rGDF11 showed no beneficial effects in dystrophic mice.
- The study suggests a potential harmful, pro-fibrotic effect of GDF11 in the context of Duchenne muscular dystrophy.
