Targeting the myostatin signaling pathway to treat muscle loss and metabolic dysfunction.

Document Type

Article

Publication Date

5-3-2021

Publication Title

The Journal of clinical investigation

Keywords

JGM

JAX Source

J Clin Invest 2021 May 3; 131(9): e148372

Volume

131

Issue

9

ISSN

1558-8238

PMID

33938454

DOI

https://doi.org/10.1172/jci148372

Grant

AR060636, AG052962

Abstract

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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