Gearing up for Preclinical Trials for Skeletal Muscle Stem Cells

SCI faculty member Rita Perlingeiro is at the forefront of research on the generation of skeletal muscle stem cells from human induced pluriptotent stem cells.  These cells could provide a much-needed therapy for individuals with skeletal muscle diseases like muscular dystrophies.  Before this type of cell therapy can be applied to humans, preclinical studies using large animals are desirable to determine potential toxicity or risks at maximal feasible dose.  In a collaboration with colleagues from the Department of Surgery, Melanie Graham and Sabarinathan Ramachandran, the Perlingeiro team has now successfully derived skeletal muscle stem cells from non-human primate (NHP) induced pluripotent stem cells.  They confirmed that these cells can engraft into muscle of mice and NHP recipients.  This study paves the way for studying the immune response to skeletal muscle cell transplants and testing human skeletal muscle stem cells in human clinical trials.  Read the published paper.