Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System
Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD ge...
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| Published in | Molecular therapy. Nucleic acids Vol. 7; no. C; pp. 11 - 19 |
|---|---|
| Main Authors | , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Elsevier Inc
16.06.2017
Elsevier Limited Elsevier American Society of Gene & Cell Therapy |
| Subjects | |
| Online Access | Get full text |
| ISSN | 2162-2531 2162-2531 |
| DOI | 10.1016/j.omtn.2017.02.004 |
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| Abstract | Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD gene by targeted deletion, and tested the efficacy of such an approach in patient-derived myogenic cells. We demonstrate restoration of wild-type dystrophin expression at transcriptional and protein level in myotubes derived from genome-edited myoblasts in the absence of selection. Removal of the duplicated exon was achieved by the use of only one guide RNA (gRNA) directed against an intronic duplicated region, thereby increasing editing efficiency and reducing the risk of off-target effects. This study opens a novel therapeutic perspective for patients carrying disease-causing duplications.
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| AbstractList | Exonic duplications account for 10%-15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD gene by targeted deletion, and tested the efficacy of such an approach in patient-derived myogenic cells. We demonstrate restoration of wild-type dystrophin expression at transcriptional and protein level in myotubes derived from genome-edited myoblasts in the absence of selection. Removal of the duplicated exon was achieved by the use of only one guide RNA (gRNA) directed against an intronic duplicated region, thereby increasing editing efficiency and reducing the risk of off-target effects. This study opens a novel therapeutic perspective for patients carrying disease-causing duplications. Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD gene by targeted deletion, and tested the efficacy of such an approach in patient-derived myogenic cells. We demonstrate restoration of wild-type dystrophin expression at transcriptional and protein level in myotubes derived from genome-edited myoblasts in the absence of selection. Removal of the duplicated exon was achieved by the use of only one guide RNA (gRNA) directed against an intronic duplicated region, thereby increasing editing efficiency and reducing the risk of off-target effects. This study opens a novel therapeutic perspective for patients carrying disease-causing duplications. [Display omitted] Exonic duplications account for 10%-15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD gene by targeted deletion, and tested the efficacy of such an approach in patient-derived myogenic cells. We demonstrate restoration of wild-type dystrophin expression at transcriptional and protein level in myotubes derived from genome-edited myoblasts in the absence of selection. Removal of the duplicated exon was achieved by the use of only one guide RNA (gRNA) directed against an intronic duplicated region, thereby increasing editing efficiency and reducing the risk of off-target effects. This study opens a novel therapeutic perspective for patients carrying disease-causing duplications. Exonic duplications account for 10%-15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD gene by targeted deletion, and tested the efficacy of such an approach in patient-derived myogenic cells. We demonstrate restoration of wild-type dystrophin expression at transcriptional and protein level in myotubes derived from genome-edited myoblasts in the absence of selection. Removal of the duplicated exon was achieved by the use of only one guide RNA (gRNA) directed against an intronic duplicated region, thereby increasing editing efficiency and reducing the risk of off-target effects. This study opens a novel therapeutic perspective for patients carrying disease-causing duplications.Exonic duplications account for 10%-15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD gene by targeted deletion, and tested the efficacy of such an approach in patient-derived myogenic cells. We demonstrate restoration of wild-type dystrophin expression at transcriptional and protein level in myotubes derived from genome-edited myoblasts in the absence of selection. Removal of the duplicated exon was achieved by the use of only one guide RNA (gRNA) directed against an intronic duplicated region, thereby increasing editing efficiency and reducing the risk of off-target effects. This study opens a novel therapeutic perspective for patients carrying disease-causing duplications. |
| Author | Bovolenta, Matteo Lattanzi, Annalisa Duguez, Stephanie Izmiryan, Araksya Bernardi, Francesco Moiani, Arianna Barbon, Elena Mamchaoui, Kamel Mouly, Vincent Mavilio, Fulvio Martin, Samia |
| AuthorAffiliation | 3 Department of Life Sciences and Biotechnology, University of Ferrara, Fossato di Mortara 74, 44121 Ferrara, Italy 1 Genethon, INSERM UMR951, 1 bis, rue de l’Internationale BP60, 91002 Evry Cedex, France 2 Institut de Myologie, UMRS 974 Sorbonne Universités UPMC-INSERM, FRE 3617 CNRS, GH Pitié-Salpétrière 47 bd de l’Hôpital, 75651 Paris Cedex 13, France |
| AuthorAffiliation_xml | – name: 2 Institut de Myologie, UMRS 974 Sorbonne Universités UPMC-INSERM, FRE 3617 CNRS, GH Pitié-Salpétrière 47 bd de l’Hôpital, 75651 Paris Cedex 13, France – name: 1 Genethon, INSERM UMR951, 1 bis, rue de l’Internationale BP60, 91002 Evry Cedex, France – name: 3 Department of Life Sciences and Biotechnology, University of Ferrara, Fossato di Mortara 74, 44121 Ferrara, Italy |
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| Copyright | 2017 The Author(s) Copyright © 2017 The Author(s). Published by Elsevier Inc. All rights reserved. 2017. The Author(s) Distributed under a Creative Commons Attribution 4.0 International License 2017 The Author(s) 2017 |
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| Keywords | dystrophin CRISPR/Cas9 gene editing duplication lentivirus |
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| Notes | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 content type line 23 Present address: University Paris Descartes Sorbonne Cité, 75015 Paris, France Present address: CNRS UMR 3215, INSERM U934, 75248 Paris, France Present address: Northern Ireland Centre for Stratified Medicine, Biomedical Sciences Research Institute, University of Ulster, C-TRIC Building, Altnagelvin Area Hospital, Glenshane Road, BT47 6SB Derry/Londonderry, Ireland Present address: INSERM UMR 1163, Imagine Institute, 75015 Paris, France Present address: Institut Curie-26, Rue d’Ulm, 75248 Paris, France |
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| Snippet | Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR... Exonic duplications account for 10%-15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR... Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR... Exonic duplications account for 10%-15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR... |
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| SubjectTerms | Biochemistry, Molecular Biology Clonal deletion CRISPR CRISPR/Cas9 Duchenne's muscular dystrophy duplication Dystrophin Gene deletion gene editing Genetics Genomes gRNA lentivirus Life Sciences Molecular biology Muscular dystrophy Mutation Myoblasts Myotubes Original Proteins Reading Ribonucleic acid RNA Transcription |
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| Title | Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System |
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