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Potential Therapeutic Action of Adiponectin in Duchenne Muscular Dystrophy
Michel Abou-Samra1, Raphaël Boursereau1, Sophie Lecompte1
1Endocrinology, Diabetes and Nutrition Unit, Institute of Experimental and Clinical Research, Medical Sector, Catholic University of Louvain, Brussels, Belgium.
Abstract:
Adiponectin (ApN) is a hormone that exhibits anti-inflammatory effects on skeletal muscle exposed to acute and chronic inflammation. We have previously tested the implication of ApN in Duchenne muscular dystrophy (DMD) using mdx mice, a model of DMD, and by generating transgenic mdx mice overexpressing ApN. We showed that ApN can act as a preventive agent and delay disease progression by reducing muscle inflammation/injury and improving force/myogenesis. Herein, we took an opposite approach and crossed mdx mice with ApN knockout mice, to obtain mdx mice with ApN depletion. The aims were to test whether ApN deficiency could worsen the mdx phenotype and whether ApN supplementation can reverse several muscle abnormalities once the disease is settled. mdx-knockout mice exhibited lower muscle force/endurance as well as increased muscle damage when compared to regular mdx mice. Local administration of the ApN gene significantly reduced the expression of several oxidative stress/inflammatory markers and increased the expression of myogenic markers in the skeletal muscle. Finally, the presence of ApN markedly reduced the activity of NF-κB, a key player in muscle inflammation and myogenesis. ApN proves to be a powerful protector of the skeletal muscle capable of reversing the disease progression, thus making it a potential therapeutic agent for DMD.
Insights
Adiponectin (ApN) deficiency worsens Duchenne muscular dystrophy (DMD) in mdx mice. However, ApN supplementation reversed muscle damage and improved function, indicating its therapeutic potential for DMD.
Area of Science:
- Biochemistry
- Molecular Biology
- Muscle Physiology
Background:
- Adiponectin (ApN) is an anti-inflammatory hormone.
- Previous studies showed ApN delays Duchenne muscular dystrophy (DMD) progression in mdx mice.
- The role of ApN deficiency and supplementation in established DMD requires further investigation.
Purpose of the Study:
- To investigate the impact of ApN depletion on the mdx mouse model of DMD.
- To evaluate the therapeutic potential of ApN supplementation in reversing DMD-related muscle abnormalities.
Main Methods:
- Crossed mdx mice with ApN knockout mice to create mdx mice with ApN depletion.
- Assessed muscle force, endurance, and damage in mdx mice with and without ApN.
- Administered ApN gene locally to mdx mice and analyzed inflammatory, oxidative stress, and myogenic markers, including NF-κB activity.
Main Results:
- ApN-deficient mdx mice showed reduced muscle force/endurance and increased muscle damage compared to regular mdx mice.
- Local ApN gene administration decreased oxidative stress/inflammatory markers and increased myogenic markers.
- ApN presence significantly reduced NF-κB activity, a key regulator of muscle inflammation and myogenesis.
Conclusions:
- ApN deficiency exacerbates the DMD phenotype in mdx mice.
- ApN acts as a potent protector of skeletal muscle, capable of reversing disease progression.
- ApN demonstrates potential as a therapeutic agent for Duchenne muscular dystrophy.
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