Target intelligence / Profile preview

Myostatin (None)

Target
None
Molecular classification
TGF-beta superfamily, Secreted protein, Growth factor
01

Overview

Myostatin, also known as growth differentiation factor 8 (GDF-8), is a secreted protein that acts as a key negative regulator of skeletal muscle growth and development. It is almost exclusively expressed in skeletal muscles, where it functions both before and after birth to limit muscle mass by inhibiting myoblast proliferation and differentiation. It signals through ActRIIB/A receptors, activating SMAD2/3 and inhibiting Akt signaling. Dysregulation contributes to muscle wasting, while inhibition is explored for treating muscle loss diseases.

Other names
GDF-8Growth Differentiation Factor 8MSTN
02

Mechanism of action

Binds activin type II receptors (ActRIIB > ActRIIA), activates SMAD2/3 signaling, inhibits Akt signaling, and promotes ubiquitin-mediated degradation of muscle proteins, ultimately inhibiting myoblast proliferation and differentiation.

03

Biological functions

Negative regulation of skeletal muscle growthInhibition of myoblast proliferationInhibition of myoblast differentiationRegulation of muscle fiber sizeRegulation of neuromuscular junction function
04

Disease associations

Muscle wasting disordersCachexiaSarcopeniaMuscular dystrophiesObesity
05

Safety considerations

Potential off-target effects on other TGF-beta signaling pathwaysUnclear long-term consequences of chronic myostatin inhibitionPossible adverse effects on cardiac muscle (although less likely)Possible effects on fertility and reproductive health
06

Interacting drugs

Follistatin

4 more in the full profile.

07

Biomarkers

Myostatin serum levelsMSTN mRNA expression in muscle tissue

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