Cell therapy is considered a potential therapeutic avenue for the treatment of skeletal muscle diseases. Heterologous approaches have been attempted in the context of Duchenne muscular dystrophy, a generalized degenerative disease. Cell transplantation trials, however, have been first hampered by the poor survival and the limited migratory ability of the cells, and a first wave of optimizations defined conditions of cell injection and recipient immunosuppression, allowing the achievement of a second set of clinical trials. Further investigations identified anoikis, oxidative stress, fusion inability and some administration methodologies as causes of early massive cell death. New concepts have emerged contemporaneously regarding the correction of gene expression (gene supplementation, exon skipping, gene surgery...), and new myogenic cell types have been identified, mainly in the family of perivascular cells, that can be administered systemically. Then, preclinical models were proposed to adapt the injection strategies, to combine them with genetic modifications of the cells or with pharmacological interventions on the environment to improve the success of implantation. In the meantime, autologous approaches have been attempted in the context of localized repairs. Therefore the continuous identification of new stem cell candidates, the invention of new strategies to restore correct gene expression, the technical and pharmacological improvements of transplantation success, are justified by yet unmet social and medical needs.