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Article

Improved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickase.

Scientific reports - 2 Dec 2016

Eggenschwiler Reto, Moslem Mohsen, Fráguas Mariane Serra, Galla Melanie, Papp Oliver, Naujock Maximilian, Fonfara Ines, Gensch Ingrid, Wähner Annabell, Beh-Pajooh Abbas, Mussolino Claudio, Tauscher Marcel, Steinemann Doris, Wegner Florian, Petri Susanne, Schambach Axel, Charpentier Emmanuelle, Cathomen Toni, Cantz Tobias

Abstract excerpt

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...

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Identifiers

PMID: 27910942
DOI: 10.1038/srep38198
PMCID: PMC5133597

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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