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Contracting CAG/CTG repeats using the CRISPR-Cas9 nickase

CAG/CTG repeat expansions cause over 13 neurological diseases that remain without a cure. Because longer tracts cause more severe phenotypes, contracting them may provide a therapeutic avenue. No currently known agent can specifically generate contractions. Using a GFP-based chromosomal reporter tha...

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Detalles Bibliográficos
Autores principales: Cinesi, Cinzia, Aeschbach, Lorène, Yang, Bin, Dion, Vincent
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5105158/
https://www.ncbi.nlm.nih.gov/pubmed/27827362
http://dx.doi.org/10.1038/ncomms13272