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

Citation metadata

Author: Se-Jin Lee
Date: May 1, 2021
From: Journal of Clinical Investigation(Vol. 131, Issue 9)
Publisher: American Society for Clinical Investigation
Document Type: Report
Length: 8,518 words
Lexile Measure: 1620L

Document controls

Main content

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.

Source Citation

Source Citation   

Gale Document Number: GALE|A661688803