Olson and colleagues generated new mouse and human iPSC models of Duchenne muscular dystrophy (DMD), harboring common exon deletions found in DMD patients. A CRISPR-Cas9 single-cut genome editing approach enabled exon skipping and exon reframing to efficiently restore dystrophin expression in the DMD models.
Min Y.-L., Chemello F., Li H., Rodriguez-Caycedo C., Sanchez-Ortiz E., Mireault A.A., et al. (2020). Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing. MOLECULAR THERAPY, 28(9), 2044-2055 [10.1016/j.ymthe.2020.05.024].
Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing
Chemello F.Co-primo
;
2020
Abstract
Olson and colleagues generated new mouse and human iPSC models of Duchenne muscular dystrophy (DMD), harboring common exon deletions found in DMD patients. A CRISPR-Cas9 single-cut genome editing approach enabled exon skipping and exon reframing to efficiently restore dystrophin expression in the DMD models.File in questo prodotto:
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Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing.pdf
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