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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Detalles Bibliográficos
Autores principales: Lo Scrudato, Mirella, Poulard, Karine, Sourd, Célia, Tomé, Stéphanie, Klein, Arnaud F., Corre, Guillaume, Huguet, Aline, Furling, Denis, Gourdon, Geneviève, Buj-Bello, Ana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6697452/
https://www.ncbi.nlm.nih.gov/pubmed/31253581
http://dx.doi.org/10.1016/j.ymthe.2019.05.021

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