CRISPR RNA-guided FokI nucleases repair a PAH variant in a phenylketonuria model

The CRISPR/Cas9 system is a recently developed genome editing technique. In this study, we used a modified CRISPR system, which employs the fusion of inactive Cas9 (dCas9) and the FokI endonuclease (FokI-dCas9) to correct the most common variant (allele frequency 21.4%) in the phenylalanine hydroxyl...

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Detalles Bibliográficos
Autores principales: Pan, Yi, Shen, Nan, Jung-Klawitter, Sabine, Betzen, Christian, Hoffmann, Georg F., Hoheisel, Jörg D., Blau, Nenad
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5081532/
https://www.ncbi.nlm.nih.gov/pubmed/27786189
http://dx.doi.org/10.1038/srep35794

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5081532/
https://www.ncbi.nlm.nih.gov/pubmed/27786189
http://dx.doi.org/10.1038/srep35794

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