Rita Perlingeiro, PhD, on the Potential of iPS Cell-based Regenerative Medicine in Muscular Dystrophy

“After all this extensive proof of principle, we have recently generated clinical grade myogenic progenitors from normal iPS [cells]. So we now have an allogeneic cell product that, if it is demonstrated safe in a first-in-human clinical trial, can theoretically be used for different types of muscular dystrophy in different patients. So this is very exciting right now for us.”

Rita Perlingeiro, PhD, the Lillehei professor in stem cell and regenerative cardiovascular medicine, Department of Medicine, University of Minnesota, recently chaired a session entitled "Using iPS Cells to Model Neuromuscular Disease" at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023. While the session was focused on disease modeling, Perlingeiro is also involved in research regarding the potential of using induced pluripotent stem (iPS) cells in the treatment of muscular dystrophy.

In an interview with CGTLive™, Perlingeiro spoke about her lab’s proof-of-concept research regarding an investigational iPS cell-derived product, noting that the cells were able to generate healthy, functional muscle when transplanted into muscular dystrophy animal models. She also mentioned that her lab has generated clinical grade allogeneic product, which she hopes will be evaluated in a clinical trial in the future. Perlingeiro concluded by reiterating the versatility of iPS cells and touched on the great progress currently occurring in general across both cell therapy and gene therapy.

Read more coverage of the 2023 MDA Conference here.

Editor’s Note: Perlingeiro disclosed that she is a co-founder of and holds equity in Myogenica Inc., a University of Minnesota start-up. She disclosed potential conflicts of interest including grants/research funding pending, grants received/research funding, patents pending, patents received, and royalties.