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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Foilsithe in:Mol Ther Nucleic Acids
Main Authors: Lattanzi, Annalisa, Duguez, Stephanie, Moiani, Arianna, Izmiryan, Araksya, Barbon, Elena, Martin, Samia, Mamchaoui, Kamel, Mouly, Vincent, Bernardi, Francesco, Mavilio, Fulvio, Bovolenta, Matteo
Formáid: Artigo
Teanga:Inglês
Foilsithe: American Society of Gene & Cell Therapy 2017
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Original Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5363679/
https://ncbi.nlm.nih.gov/pubmed/28624187
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.02.004
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