Functional improvement of dystrophic muscle by myostatin blockade

Sasha Bogdanovich, Thomas O.B. Krag, Elisabeth R. Barton, Linda D. Morris, Lisa Anne Whittemore, Rexford S. Ahima, Tejvir S. Khurana*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

675 Scopus citations

Abstract

Mice and cattle with mutations in the myostatin (GDF8) gene show a marked increase in body weight and muscle mass, indicating that this new member of the TGF-β superfamily is a negative regulator of skeletal muscle growth. Inhibition of the myostatin gene product is predicted to increase muscle mass and improve the disease phenotype in a variety of primary and secondary myopathies. We tested the ability of inhibition of myostatin in vivo to ameliorate the dystrophic phenotype in the mdx mouse model of Duchenne muscular dystrophy (DMD). Blockade of endogenous myostatin by using intraperitoneal injections of blocking antibodies for three months resulted in an increase in body weight, muscle mass, muscle size and absolute muscle strength in mdx mouse muscle along with a significant decrease in muscle degeneration and concentrations of serum creatine kinase. The functional improvement of dystrophic muscle by myostatin blockade provides a novel, pharmacological strategy for treatment of diseases associated with muscle wasting such as DMD, and circumvents the major problems associated with conventional gene therapy in these disorders.

Original languageEnglish (US)
Pages (from-to)418-421
Number of pages4
JournalNature
Volume420
Issue number6914
DOIs
StatePublished - Nov 28 2002

ASJC Scopus subject areas

  • General

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