CRISPR–Cas9 paired with adeno-associated virus serotype 6 (AAV6) is among the most efficient tools for producing targeted gene knockins. Here, we report that this system can lead to frequent concatemeric insertions of the viral vector genome at the target site that are difficult to detect.
CRISPR–Cas9 paired with adeno-associated virus serotype 6 is among the most efficient tools for producing targeted gene knockins. Here, we report that this system can lead to frequent concatemeric insertions of the viral vector genome at the target site that are difficult to detect. Such errors can cause adverse and unreliable phenotypes that are antithetical to the goal of precision genome engineering.
Itoh, M. et al. Footprint-free gene mutation correction in induced pluripotent stem cell derived from recessive dystrophic epidermolysis bullosa using the CRISPR/Cas9 andWilkinson, A. C. et al. Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice.Martin, R. M. et al. Highly efficient and marker-free genome editing of human pluripotent stem cells by CRISPR–Cas9 RNP and AAV6 donor-mediated homologous recombination.Romero, Z. et al.
Fabian P. Suchy, Daiki Karigane, Yusuke Nakauchi, Maimi Higuchi, Jinyu Zhang, Ian Hsu, Amy C. Fan, Toshinobu Nishimura, Carsten T. Charlesworth, Joydeep Bhadury, Toshiya Nishimura, Adam C. Wilkinson, Ravindra Majeti & Hiromitsu NakauchiFabian P. Suchy, Maimi Higuchi, Jinyu Zhang, Katja Pekrun, Ian Hsu, Toshinobu Nishimura, Carsten T. Charlesworth, Joydeep Bhadury, Toshiya Nishimura, Adam C. Wilkinson, Mark A. Kay & Hiromitsu NakauchiDaiki Karigane, Yusuke Nakauchi, Amy C.
, Schematic indicating ddPCR linkage analysis strategy for measuring KI frequency in bulk samples. Cis reference sites shown as small light-gray and dark-gray squares. Ubc-GFP amplicon site indicated by small green square. Dashed-lines indicate linkage, which can be measured by ddPCR. ID1 and ID2 are small unique DNA sequences added outside the homology arms in the viral vector genome as shown at the top.
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