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Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells
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A new era for myotonic dystrophy: Improved delivery of a DMPK-targeted oligonucleotide modulates muscle

Eric T Wang1

  • 1Department of Molecular Genetics & Microbiology, Center for NeuroGenetics, University of Florida, Gainesville, FL, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|June 24, 2026
PubMed
Abstract

No abstract available in PubMed .

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Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...

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