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Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy

Duchenne muscular dystrophy is caused by mutations in DMD which disrupt the reading frame. Therapeutic strategies that restore DMD’s reading frame, such as exon skipping and CRISPR/Cas9, need to be tested in the context of the human DMD sequence in vivo. We have developed a novel dystrophic mouse mo...

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Détails bibliographiques
Publié dans:J Neuromuscul Dis
Auteurs principaux: Young, Courtney S., Mokhonova, Ekaterina, Quinonez, Marbella, Pyle, April D., Spencer, Melissa J.
Format: Artigo
Langue:Inglês
Publié: 2017
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5565771/
https://ncbi.nlm.nih.gov/pubmed/28505980
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3233/JND-170218
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