CD133+ cells derived from skeletal muscles of Duchenne muscular dystrophy patients have a compromised myogenic and muscle regenerative capability

Stem Cell Research
Jinhong MengJennifer Morgan

Abstract

Cell-mediated gene therapy is a possible means to treat muscular dystrophies like Duchenne muscular dystrophy. Autologous patient stem cells can be genetically-corrected and transplanted back into the patient, without causing immunorejection problems. Regenerated muscle fibres derived from these cells will express the missing dystrophin protein, thus improving muscle function. CD133+ cells derived from normal human skeletal muscle contribute to regenerated muscle fibres and form muscle stem cells after their intra-muscular transplantation into an immunodeficient mouse model. But it is not known whether CD133+ cells derived from DMD patient muscles have compromised muscle regenerative function. To test this, we compared CD133+ cells derived from DMD and normal human muscles. DMD CD133+ cells had a reduced capacity to undergo myogenic differentiation in vitro compared with CD133+ cells derived from normal muscle. In contrast to CD133+ cells derived from normal human muscle, those derived from DMD muscle formed no satellite cells and gave rise to significantly fewer muscle fibres of donor origin, after their intra-muscular transplantation into an immunodeficient, non-dystrophic, mouse muscle. DMD CD133+ cells gave rise to more clo...Continue Reading

Citations

May 3, 2019·PloS One·Mona BensalahElisa Negroni
Aug 9, 2019·Journal of Muscle Research and Cell Motility·Amber L Mueller, Robert J Bloch
Sep 19, 2020·The Journal of Clinical Investigation·Stefano BiressiThomas A Rando
Aug 20, 2021·Frontiers in Genetics·Olivier BoyerUNKNOWN Study Group

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Methods Mentioned

BETA
biopsies
biopsy
FACS

Software Mentioned

Graphpad Prism5
MetaMorph

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