Completed Preclinical (in vivo) 2009

    Follistatin gene delivery enhances muscle growth and strength in nonhuman primates

    Kota J, Handy CR, Haidet AM, et al.

    Science Translational Medicine

    DOI: 10.1126/scitranslmed.3000112

    Summary

    Demonstrated that AAV-mediated follistatin gene delivery produced significant and sustained increases in muscle mass and strength in nonhuman primates. This was the first primate study to confirm follistatin's myostatin-inhibiting muscle growth effects translate beyond rodent models.

    Key Findings

    • Follistatin gene delivery increased muscle size and strength in cynomolgus macaques
    • Effects were sustained over long-term follow-up without adverse effects
    • First primate validation of follistatin's muscle-enhancing properties

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    Completed 2009

    Follistatin induces muscle hypertrophy through satellite cell proliferation and inhibition of both myostatin and activin

    Gilson H, Schakman O, Kalista S, et al.

    American Journal of Physiology-Endocrinology and Metabolism

    Revealed that follistatin-induced muscle hypertrophy occurs through dual mechanisms: satellite cell proliferation and simultaneous inhibition of both myostatin and activin signaling. Demonstrated that follistatin's effects extend beyond simple myostatin antagonism.

    • Follistatin induced significant muscle hypertrophy through satellite cell activation
    • Inhibited both myostatin and activin, not myostatin alone

    DOI: 10.1152/ajpendo.91073.2008

    Completed 2001

    Regulation of myostatin activity and muscle growth

    Lee SJ, McPherron AC

    Proceedings of the National Academy of Sciences

    Foundational study establishing that follistatin is a potent endogenous inhibitor of myostatin. Transgenic mice overexpressing follistatin exhibited dramatic increases in muscle mass exceeding those seen in myostatin knockout animals, suggesting follistatin inhibits additional negative regulators of muscle growth.

    • Follistatin overexpression produced muscle mass increases exceeding myostatin knockout alone
    • Established follistatin as a natural myostatin antagonist in vivo

    DOI: 10.1073/pnas.151270098