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Updated: Jul 19, 2026

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
Published on: September 14, 2019
Stem cell based therapies to treat muscular dystrophy
F D Price1, K Kuroda, M A Rudnicki
1Molecular Medicine Program and Center for Stem Cell and Gene Therapy, Ottawa Health Research Institute, 501 Smyth Road, Ottawa, ON, Canada K1H 8L6.
Abstract:
Muscular dystrophies comprise a heterogeneous group of neuromuscular disorders, characterized by progressive muscle wasting, for which no satisfactory treatment exists. Multiple stem cell populations, both of adult or embryonic origin, display myogenic potential and have been assayed for their ability to correct the dystrophic phenotype. To date, many of these described methods have failed, underlying the need to identify the mechanisms controlling myogenic potential, homing of donor populations to the musculature, and avoidance of the immune response. Recent results focus on the fresh isolation of satellite cells and the use of multiple growth factors to promote mesangioblast migration, both of which promote muscle regeneration. Throughout this chapter, various stem cell based therapies will be introduced and evaluated based on their potential to treat muscular dystrophy in an effective and efficient manner.
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