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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:F1000Res
Prif Awduron: Bhagwan, Jamie R., Collins, Emma, Mosqueira, Diogo, Bakar, Mine, Johnson, Benjamin B., Thompson, Alexander, Smith, James G.W., Denning, Chris
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: F1000 Research Limited 2020
Pynciau:
Research Article
Mynediad Ar-lein: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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