Aim: Muscle-based somatic gene therapy is a novel way to alleviate a bioche
mical deficiency. Method: Muscle-derived cells are very promising in the fi
eld of gene therapy and tissue engineering. First, most muscle tissue is ac
cessible by injection. Second, muscle tissue consists of multinucleated, po
stmitotic myofibers, which enable a long-term expression of the transduced
gene. Third, muscle tissue can be biopsied easily. It is available in abund
ance and the biopsy does not compromise the health and function of the pati
ent. Finally, muscle tissue is highly vascularized, which makes systemic de
livery feasible. Results: Muscle-derived cells can promote muscle healing a
nd bone healing. implanted cells maintain a long-term transgene expression
of therapeutic proteins. Isolated, muscle-derived stem cells can differenti
ate in osteoblasts. Conclusion: Based on these characteristics, we present
four possible applications: inherited muscular diseases, muscle injury, bon
e healing, and intraarticular disorders.