Clinical Application of Induced Pluripotent Stem Cells in Muscular Dystrophy: Rita Perlingeiro, PhD

WATCH TIME: 5 minutes

“We have demonstrated in many studies that the (induced pluripotent stem [iPS]) cells must be transplanting animal models of muscular dystrophy. We are able to generate healthy muscle that's functional, and the cells are able also to see the stem cell, which is very important for long term regeneration.”

Using patient-derived induced pluripotent stem (iPS) cells, different pathogenesis of neuromuscular diseases have been analyzed, leading toward significant progress in screening for treatment. For example, in amyotrophic lateral sclerosis, researchers have performed high throughput screening using iPS cells-obtained in motor neurons to identify potential candidate drugs.¹

At the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, Rita Perlingeiro, PhD, recently presented a talk on the potential of using iPS cells in the treatment of muscular dystrophy. The rest of the session focused on the use of patient-specific iPS cells to model neuromuscular diseases, with the goal of understanding disease pathogenesis and treatment development. Also, the session emphasized the expansion of bioengineering approaches for skeletal muscle modeling.²

In an interview with CGTLive®, a sister publication to NeurologyLive®, Perlingeiro, the Lillehei professor in stem cell and regenerative cardiovascular medicine, department of medicine, University of Minnesota, provided an overview of some of the main highlights addressed in the session. She also spoke about the challenges and unmet needs in the field starting to gain attention, as well as the potential of iPS in regenerative medicine.

Click here for more coverage of MDA 2023.

REFERENCES
1. Ito D, Morimoto S, Takahashi S, Okada K, Nakahara J, Okano H. Maiden voyage: induced pluripotent stem cell-based drug screening for amyotrophic lateral sclerosis. Brain. 2023;146(1):13-19. doi:10.1093/brain/awac306
2. Perlingeiro R. Using patient-specific iPS cells to model and target FKRP mutations. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Using iPS Cells to Model Neuromuscular Disease session.

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.