1The Jackson Laboratory for Genomic Medicine, Farmington, Connecticut, USA.
2University of Connecticut School of Medicine, Department of Genetics and Genome Sciences, Farmington, Connecticut, USA.
Address correspondence to: Se-Jin Lee, The Jackson Laboratory, 10 Discovery Drive, Farmington, Connecticut 06032, USA; Phone: 860.837.2183; Email: Se-Jin.Lee@jax.org.
Published May 3, 2021 - More info
Since the discovery of myostatin (MSTN; also known as GDF-8) as a critical regulator of skeletal muscle mass in 1997, there has been an extensive effort directed at understanding the cellular and physiological mechanisms underlying MSTN activity, with the long-term goal of developing strategies and agents capable of blocking MSTN signaling to treat patients with muscle loss. Considerable progress has been made in elucidating key components of this regulatory system, and in parallel with this effort has been the development of numerous biologics that have been tested in clinical trials for a wide range of indications, including muscular dystrophy, sporadic inclusion body myositis, spinal muscular atrophy, cachexia, muscle loss due to aging or following falls, obesity, and type 2 diabetes. Here, I review what is known about the MSTN regulatory system and the current state of efforts to target this pathway for clinical applications.
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