**1. Introduction**

Muscular dystrophies (MDs) are genetic diseases caused by the continuous degeneration/ regeneration cycles of skeletal muscle tissue. Mutations in genes encoding for proteins, either at the plasma membrane or within internal membrane, are responsible for MDs. During contractions, the affected muscle fibres degenerate and the molecular mechanisms are not yet fully understood. Fibre loss is compensated by the regeneration of new fibres, mainly sustained by satellite cells. These are localized underneath the basal lamina of muscle fibres [1]. Damaged dystrophic muscles engage in a remodelling process to generate novel fibres and to produce abundant extracellular matrix (ECM). ECM is necessary for adequate tissue repair. During periods of degeneration/regeneration, myofibroblasts accumulate in dystrophic muscles and are responsible for large amounts of extracellular matrix proteins, generating fibrosis. Addi‐ tionally, at the final stage, satellite cells become exhausted and are not able to generate new fibres. Cardiac muscle is less efficient in regeneration, compared to skeletal muscle and scar tissues in replacing damaged cardiomyocytes after injuries [2]. However, several research groups have demonstrated the presence of stem/progenitor cells that are able to differentiate into cardiac tissues [3-6], as well as skeletal muscle lineages [7-12]. This paper deals with novel therapeutic approaches for skeletal muscle dystrophies and explores pharmacological treatments. It also provides more recent gene and cell therapeutic protocols. Different sources of myogenic stem cells are discussed, highlighting their advantages and disadvantages, as well as underlining controversies in literature. Finally, we discuss autologous and heterologous cell therapy, considering the viral and non-viral technologies for *ex vivo* cell therapy in the treatment of muscular dystrophies.
