Muscular dystrophies (MD) are heterogeneous group of genetic disorders including more than 30 different types [1,2]. They are generally characterized by progressive degeneration and fibrosis of the skeletal muscles. These disorders are mostly due to gene defects in important structural proteins in muscle which lead to muscle tissue damage and eventually partial or complete loss of muscle function with various morbidity or mortality [3]. Though in many cases the defective genes are known, there is no definitive cure so far. Meanwhile gene delivery or stem cell based therapies are promising approaches to correct the gene defect or restore the skeletal muscle tissue with healthy stem cells which provide the correct genetic information to the muscle [4]. However, except finding the right type of stem cells to restore the muscle, one of the major hurdles in stem cell therapy is the route of cell delivery into the dystrophic muscle. Due to complexity of the muscle groups involved in different muscular dystrophies, local cell delivery is not a feasible and practical approach [5-7]. Therefore, ideally the cells needed to be administered systemically to reach multiple involved muscle groups. These editorial overviews common types of muscular dystrophies, different stem cell populations tested for systemic delivery/engraftment into skeletal muscle and outlines key factors and challenges for successful systemic cell delivery, migration and engraftment into skeletal muscle
Author(s): Nadine Matthias and Radbod Darabi.