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santé - iate.europa.eu
santé - iate.europa.eu
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... We show that this strategy indeed not only permits bi-allelic seamless repair of the beta-globin gene splice site mutation and negligible off-target mutagenesis or re-editing of the targeted allele but also results in unexpected on-target mutagenesis with some guide RNAs ...
général - core.ac.uk - PDF: doaj.org


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