In vivo CRISPR-Cas gene editing with no detectable genome-wide off-target mutations

Preprint OPEN
Akcakaya, Pinar; Bobbin, Maggie; Guo, Jimmy; Lopez, Jose; Clement, M.; Garcia, Sara; Fellows, Mick; Porritt, Michelle; Firth, Mike; Carreras, Alba; Baccega, Tania; Seeliger, Frank; Bjursell, Mikael; Tsai, Shengdar; Nguyen, Nhu; Nitsch, Roberto; Mayr, Lorenz; Pinello, Luca; Bohlooly-Y, Mohammad; Aryee, Martin; Maresca, Marcello; Joung, J.;
(2018)

CRISPR-Cas genome-editing nucleases hold substantial promise for human therapeutics but identifying unwanted off-target mutations remains an important requirement for clinical translation. For ex vivo therapeutic applications, previously published cell-based genome-wide... View more
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