Variable expression and silencing of CRISPR-Cas9 targeted transgenes identifies the AAVS1 locus as not an entirely safe harbour

Background: Diseases such as hypertrophic cardiomyopathy (HCM) can lead to severe outcomes including sudden death. The generation of human induced pluripotent stem cell (hiPSC) reporter lines can be useful for disease modelling and drug screening by providing physiologically relevant in vitro models...

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Detalles Bibliográficos
Autores principales: Bhagwan, Jamie R., Collins, Emma, Mosqueira, Diogo, Bakar, Mine, Johnson, Benjamin B., Thompson, Alexander, Smith, James G.W., Denning, Chris
Formato: Online Artículo Texto
Lenguaje:English
Publicado: F1000 Research Limited 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7401084/
https://www.ncbi.nlm.nih.gov/pubmed/32789000
http://dx.doi.org/10.12688/f1000research.19894.2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7401084/
https://www.ncbi.nlm.nih.gov/pubmed/32789000
http://dx.doi.org/10.12688/f1000research.19894.2

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