STUDIES ON ADIPOSE AND MUSCLE-DERIVED STEM CELLS AS TREATMENT FOR MUSCULAR DYSTROPHY INDUCED IN RATS

Abstract

Muscular dystrophy is a life threatening disorder that affects the skeletal muscles due to a mutation on the dystrophin gene which is located on chromosome Xp21 and encodes for the dystrophin protein. Dystrophin gene mutations lead to synthesis of an internally truncated or partially functional dystrophin protein which is associated with variable phenotypes of muscular dystrophy. Nine major forms of muscular dystrophy are known and differ clinically in many aspects including age of onset, spectrum of muscle groups affected, severity of muscle injury and lethality.

The dystrophin has crucial roles in myofiber stability and integrity by connecting the actin cytoskeleton to the extracellular matrix, but unfortunately, lack of dystrophin leads to myofiber fragility and contributes to skeletal muscle degeneration in muscular dystrophy patients with the production of abundant fibrous and adipose tissue in the later stages of the disorder. Muscular dystrophy can also lead to loss of motor functions in puberty with a great possibility of losing cardiac muscle and respiratory functions and hence, premature death.