Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice

Myotonic dystrophy type 1 (DM1) is caused by a CTG repeat expansion located in the 3′ UTR of the DMPK gene. Expanded DMPK transcripts aggregate into nuclear foci and alter the function of RNA-binding proteins, leading to defects in the alternative splicing of numerous pre-mRNAs. To date, there is no...

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Publicat a:Mol Ther
Autors principals: Lo Scrudato, Mirella, Poulard, Karine, Sourd, Célia, Tomé, Stéphanie, Klein, Arnaud F., Corre, Guillaume, Huguet, Aline, Furling, Denis, Gourdon, Geneviève, Buj-Bello, Ana
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2019
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6697452/
https://ncbi.nlm.nih.gov/pubmed/31253581
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.05.021
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