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Expert Opin Biol Ther. 2006 Feb;6(2):147-54.

The role of myostatin and bone morphogenetic proteins in muscular

disorders.

Tsuchida K.

Institute for Comprehensive Medical Science, Division for Therapies

against Intractable Diseases, Fujita Health University, Toyoake,

Aichi 470-1192, Japan.

Skeletal muscle is the largest organ in the human body, and plays an

important role in body movement and metabolism. Skeletal muscle mass

is lost in genetic disorders such as muscular dystrophy, muscle

wasting and ageing. Chemicals and proteins that restore muscle mass

and function are potential drugs that can improve human health and

could be used in the clinic.

Myostatin is a muscle-specific member of the transforming growth

factor (TGF)-beta superfamily that plays an essential role in the

negative regulation of muscle growth. Inhibition of myostatin

activity is a promising therapeutic method for restoring muscle mass

and strength. Potential inhibitors of myostatin include follistatin

domain-containing proteins, myostatin propeptide, myostatin

antibodies and chemical compounds. These inhibitors could be

beneficial for the development of clinical drugs for the treatment of

muscular disorders. Bone morphogenetic protein (BMP) plays a

significant role in the development of neuromuscular architecture and

its proper functions. Modulation of BMP activity could be beneficial

for muscle function in muscular disorders. This review will describe

the current progress in therapy for muscular disorders, emphasising

the importance of myostatin as a drug target.

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