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Updated: Oct 3, 2025

Preparation of Primary Myogenic Precursor Cell/Myoblast Cultures from Basal Vertebrate Lineages
Published on: April 30, 2014
Myostatin: Basic biology to clinical application
Pasquale Esposito1, Daniela Picciotto1, Yuri Battaglia2
1Clinica Nefrologica, Dialisi, Trapianto, Department of Internal Medicine, University of Genoa and IRCCS Ospedale Policlinico San Martino, Genova, Italy.
Abstract:
Myostatin is a member of the transforming growth factor (TGF)-β superfamily. It is expressed by animal and human skeletal muscle cells where it limits muscle growth and promotes protein breakdown. Its effects are influenced by complex mechanisms including transcriptional and epigenetic regulation and modulation by extracellular binding proteins. Due to its actions in promoting muscle atrophy and cachexia, myostatin has been investigated as a promising therapeutic target to counteract muscle mass loss in experimental models and patients affected by different muscle-wasting conditions. Moreover, growing evidence indicates that myostatin, beyond to regulate skeletal muscle growth, may have a role in many physiologic and pathologic processes, such as obesity, insulin resistance, cardiovascular and chronic kidney disease. In this chapter, we review myostatin biology, including intracellular and extracellular regulatory pathways, and the role of myostatin in modulating physiologic processes, such as muscle growth and aging. Moreover, we discuss the most relevant experimental and clinical evidence supporting the extra-muscle effects of myostatin. Finally, we consider the main strategies developed and tested to inhibit myostatin in clinical trials and discuss the limits and future perspectives of the research on myostatin.
Insights
Myostatin, a protein limiting muscle growth, is a therapeutic target for muscle wasting. Research explores its broader roles in obesity, cardiovascular, and kidney diseases, alongside inhibition strategies.
Area of Science:
- Biochemistry
- Molecular Biology
- Physiology
Background:
- Myostatin, a TGF-β superfamily member, regulates skeletal muscle mass by limiting growth and promoting protein breakdown.
- Its expression and activity are controlled by complex transcriptional, epigenetic, and extracellular binding protein mechanisms.
- Myostatin's role in muscle atrophy and cachexia makes it a therapeutic target for muscle-wasting conditions.
Purpose of the Study:
- To review myostatin biology, including its regulatory pathways.
- To discuss the evidence for myostatin's extra-muscular effects in various physiological and pathological processes.
- To examine therapeutic strategies targeting myostatin inhibition.
Main Methods:
- Literature review of myostatin biology and its regulatory pathways.
- Analysis of experimental and clinical evidence on myostatin's role beyond skeletal muscle.
- Evaluation of current and past clinical trials for myostatin inhibitors.
Main Results:
- Myostatin influences skeletal muscle growth, aging, obesity, insulin resistance, cardiovascular, and chronic kidney disease.
- Various strategies to inhibit myostatin have been developed and tested in clinical trials.
- Evidence supports myostatin's significant role in diverse physiological and pathological processes.
Conclusions:
- Myostatin is a key regulator of muscle mass with implications for numerous diseases.
- Inhibiting myostatin presents therapeutic potential but requires careful consideration of its multifaceted roles.
- Further research is needed to fully understand and harness myostatin's therapeutic potential.
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