Figure 1.

Potential protocol for improving cell therapy for muscular dystrophy. With advancements in the isolation and culture of muscle stem cells, the following may become possible. Skeletal muscle satellite cells (SCs) could be obtained by muscle biopsy or from cadaver muscle and enzymatically disaggregated to a single cell suspension containing an impure population of satellite cells. Satellite stem cells could be purified by flow cytometry. Alternatively, satellite cells could be derived from reprogrammed iPSCs. Culture conditions that allow the expansion of only the stem cell subpopulation of satellite cells would improve transplantation and require only limited cell numbers (e.g. the use of hydrogels and low levels of oxygen). Genetic correction of autologous satellite cells would also be required. Preclinical studies in animal models, such as the dystrophin deficient mdx mouse and golden retriever muscular dystrophy dog, would be performed to confirm safety and efficacy before the therapy enters the clinic. Currently, satellite cells are only deliverable intramuscularly, although further understanding of their biology may allow their modification so that they can be delivered systemically.