CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo

Duchenne muscular dystrophy (DMD), a severe hereditary disease affecting 1 in 3,500 boys, mainly results from the deletion of exon(s), leading to a reading frameshift of the DMD gene that abrogates dystrophin protein synthesis. Pairs of sgRNAs for the Cas9 of Staphylococcus aureus were meticulously...

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Bibliografske podrobnosti
izdano v:Mol Ther
Main Authors: Duchêne, Benjamin L., Cherif, Khadija, Iyombe-Engembe, Jean-Paul, Guyon, Antoine, Rousseau, Joel, Ouellet, Dominique L., Barbeau, Xavier, Lague, Patrick, Tremblay, Jacques P.
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2018
Teme:
Original Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6224775/
https://ncbi.nlm.nih.gov/pubmed/30195724
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.08.010
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