Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing

Nonsense-mediated decay (NMD) is a major pathogenic mechanism underlying a diversity of genetic disorders. Nonsense variants tend to lead to more severe disease phenotypes and are often difficult targets for small molecule therapeutic development as a result of insufficient protein production. The t...

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Detalles Bibliográficos
Autores principales: Erwood, Steven, Laselva, Onofrio, Bily, Teija M.I., Brewer, Reid A., Rutherford, Alexandra H., Bear, Christine E., Ivakine, Evgueni A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7256445/
https://www.ncbi.nlm.nih.gov/pubmed/32490033
http://dx.doi.org/10.1016/j.omtm.2020.05.002

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