Targeting myostatin for therapies against muscle-wasting disorders

[Guest guest]

Curr Opin Drug Discov Devel. 2008 Jul;11(4):487-94.

Targeting myostatin for therapies against muscle-wasting disorders.

Tsuchida K.

Division for Therapies against Intractable Diseases, Institute for

Comprehensive Medical Science (ICMS), Fujita Health University,

Toyoake, Aichi 470-1192, Japan.

In addition to gene correction therapy and cell transplantation

techniques, multidisciplinary approaches to drug discovery and

development offer promising therapeutic strategies for intractable

genetic muscular disorders including muscular dystrophy.

Inhibition of the production and activity of myostatin, a potent

growth factor that determines skeletal muscle size, is a novel

strategy for the treatment of muscle-wasting disorders such as

muscular dystrophy, cachexia and sarcopenia.

Myostatin blockers include myostatin-blocking antibodies, myostatin

propeptide, follistatin and follistatin-related proteins, soluble

myostatin receptors, small interfering RNA and small chemical

inhibitors.

This review describes the discovery and development of myostatin

inhibitors.