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Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles

Gene editing is often touted as a permanent method for correcting mutations, but its long-term benefits in Duchenne muscular dystrophy (DMD) may depend on sufficiently high editing efficiencies to halt muscle degeneration. Here, we explored the persistence of dystrophin expression following recombin...

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Foilsithe in:Mol Ther
Main Authors: Bengtsson, Niclas E., Tasfaout, Hichem, Hauschka, Stephen D., Chamberlain, Jeffrey S.
Formáid: Artigo
Teanga:Inglês
Foilsithe: American Society of Gene & Cell Therapy 2021
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7934576/
https://ncbi.nlm.nih.gov/pubmed/33160075
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.11.003
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