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Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

The CRISPR/Cas9 system is a great revolution in biology. This technology allows the modification of genes in vitro and in vivo in a wide variety of living organisms. In most Duchenne muscular dystrophy (DMD) patients, expression of dystrophin (DYS) protein is disrupted because exon deletions result...

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Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: Iyombe-Engembe, Jean-Paul, Ouellet, Dominique L, Barbeau, Xavier, Rousseau, Joël, Chapdelaine, Pierre, Lagüe, Patrick, Tremblay, Jacques P
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5012554/
https://ncbi.nlm.nih.gov/pubmed/26812655
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2015.58
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