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.
Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing / Min Y.-L.; Chemello F.; Li H.; Rodriguez-Caycedo C.; Sanchez-Ortiz E.; Mireault A.A.; McAnally J.R.; Shelton J.M.; Zhang Y.; Bassel-Duby R.; Olson E.N.. - In: MOLECULAR THERAPY. - ISSN 1525-0016. - ELETTRONICO. - 28:9(2020), pp. 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 | Dimensione | Formato | |
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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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