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Development of a novel gene editing lexicon for hemophilia: methodology and results Kessler, Craig M.; Valentino, Leonard A.; Thornburg, Courtney D.; Unzu, Carmen; Kay, Mark A.; Peyvandi, Flora; Smith, Penni; Miesbach, Wolfgang; Mckeown, William; Pierce, Glenn F.; Khair, Kate; Starcevic, Katarina; Pillai, Monisha; Jones, Micheala; Sindhurakar, Anil; Whyte, Lauren; Delwart, Virginie; Chiao, Megan; Gutstein, David E.; Antonino, Ilia; Hermans, Cedric; Pipe, Steven W. Share Save
Operationalising a Haemophilia Gene Editing Lexicon for Practical Use Mckeown, William; Hermans, Cedric; Unzu, Carmen; Kay, Mark A.; Peyvandi, Flora; Smith, Penni; Miesbach, Wolfgang; Pierce, Glenn F.; Khair, Kate; Valentino, Leonard A.; Pipe, Steven W.; Pillai, Monisha; Jones, Micheala; Delwart, Virginie; Sindhurakar, Anil; Gutstein, David E.; Kessler, Craig M. Share Save
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A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results Hermans, Cedric; Valentino, Leonard A.; Thornburg, Courtney D.; Unzu, Carmen; Kay, Mark A.; Peyvandi, Flora; Smith, Penni; Miesbach, Wolfgang; Mckeown, William; Pierce, Glenn F.; Khair, Kate; Pipe, Steven W.; Starcevic, Katarina; Pillai, Monisha; Jones, Micheala; Chiao, Megan; Antonino, Ilia; Kessler, Craig Share Save
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Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectors Suchy, Fabian P.; Karigane, Daiki; Nakauchi, Yusuke; Higuchi, Maimi; Zhang, Jinyu; Pekrun, Katja; Hsu, Ian; Fan, Amy C.; Nishimura, Toshinobu; Charlesworth, Carsten T.; Bhadury, Joydeep; Nishimura, Toshiya; Wilkinson, Adam C.; Kay, Mark A.; Majeti, Ravindra; Nakauchi, Hiromitsu Share Save
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A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors Davis-Gardner, Meredith E.; Weber, Jesse A.; Xie, Jun; Pekrun, Katja; Alexander, Eric A.; Weisgrau, Kim L.; Furlott, Jessica R.; Rakasz, Eva G.; Kay, Mark A.; Gao, Guangping; Farzan, Michael; Gardner, Matthew R. Share Save
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