Ameliorated cellular hallmarks of myotonic dystrophy in hybrid myotubes from patient and unaffected donor cells.
Renée H L RaaijmakersC Rosanne M AusemsMarieke WillemseSarah A CummingBaziel G M van EngelenDarren G MoncktonHans van BokhovenDerick G WansinkPublished in: Stem cell research & therapy (2024)
Fusion of only a limited number of unaffected myogenic precursors to DM1 myotubes already ameliorates cellular disease hallmarks, offering promise for the development of cell transplantation strategies to lower disease burden.