Identification of on-target mutagenesis during correction of a beta-thalassemia splice mutation in iPS cells with optimised CRISPR/Cas9-double nickase reveals potential safety concerns

Precise and accurate gene correction is crucial for enabling iPSC-based therapies, and Cas9-Nickase based approaches are increasingly considered for in vivo correction of diseases such as beta-thalassemia. Here, we generate footprint-free induced pluripotent stem cells from a patient with a beta-tha...

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Detalles Bibliográficos
Autores principales: Alateeq, Suad, Ovchinnikov, Dmitry, Tracey, Timothy, Whitworth, Deanne, Al-Rubaish, Abdullah, Al-Ali, Amein, Wolvetang, Ernst
Formato: Online Artículo Texto
Lenguaje:English
Publicado: AIP Publishing LLC 2018
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6481731/
https://www.ncbi.nlm.nih.gov/pubmed/31069325
http://dx.doi.org/10.1063/1.5048625

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6481731/
https://www.ncbi.nlm.nih.gov/pubmed/31069325
http://dx.doi.org/10.1063/1.5048625

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