Ontology highlight
ABSTRACT:
SUBMITTER: Eggenschwiler R
PROVIDER: S-EPMC5133597 | biostudies-literature | 2016 Dec
REPOSITORIES: biostudies-literature
Eggenschwiler Reto R Moslem Mohsen M Fráguas Mariane Serra MS Galla Melanie M Papp Oliver O Naujock Maximilian M Fonfara Ines I Gensch Ingrid I Wähner Annabell A Beh-Pajooh Abbas A Mussolino Claudio C Tauscher Marcel M Steinemann Doris D Wegner Florian F Petri Susanne S Schambach Axel A Charpentier Emmanuelle E Cathomen Toni T Cantz Tobias T
Scientific reports 20161202
Homology directed repair (HDR)-based genome editing via selectable long flanking arm donors can be hampered by local transgene silencing at transcriptionally silent loci. Here, we report efficient bi-allelic modification of a silent locus in patient-derived hiPSC by using Cas9 nickase and a silencing-resistant donor construct that contains an excisable selection/counter-selection cassette. To identify the most active single guide RNA (sgRNA)/nickase combinations, we employed a lentiviral vector- ...[more]