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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Detalhes bibliográficos
Publicado no:F1000Res
Main Authors: Bhagwan, Jamie R., Collins, Emma, Mosqueira, Diogo, Bakar, Mine, Johnson, Benjamin B., Thompson, Alexander, Smith, James G.W., Denning, Chris
Formato: Artigo
Idioma:Inglês
Publicado em: F1000 Research Limited 2020
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7401084/
https://ncbi.nlm.nih.gov/pubmed/32789000
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.12688/f1000research.19894.2
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