Intramuscular cell transplantation as a potential treatment of myopathies: clinical and preclinical relevant data

Myopathies produce deficits in skeletal muscle function and, in some cases, literally progressive loss of skeletal muscles. The transplantation of cells able to differentiate into myofibers is an experimental strategy for the potential treatment of some of these diseases.

Among the two routes used to deliver cells to skeletal muscles, that is intramuscular and intravascular, this paper focuses on the intramuscular route due to our expertise and because it is the most used in animal experiments and the only tested so far in humans. Given the absence of recent reviews about clinical observations and the profusion based on mouse results, this review prioritizes observations made in humans and non-human primates. The review provides a vision of cell transplantation in myology centered on what can be learned from clinical trials and from preclinical studies in non-human primates and leading mouse studies.

Experiments on myogenic cell transplantation in mice are essential to quickly identify potential treatments, but studies showing the possibility to scale up the methods in large mammals are indispensable to determine their applicability in humans and to design clinical protocols.