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In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

Kiran Musunuru, Alexandra C. Chadwick, Taiji Mizoguchi, Sara P. Garcia, Jamie E. DeNizio, C.W. Reiss, Kui Wang, Sowmya Iyer, Chaitali Dutta, Victoria Clendaniel, Michael Amaonye, Aaron Beach, Kathleen Berth, Souvik Biswas, Maurine Braun, Huei-Mei Chen, Thomas V. Colace, John D. Ganey, Soumyashree A. Gangopadhyay, Ryan Garrity, Lisa N. Kasiewicz, Jennifer Lavoie, James A. Madsen, Yuri Matsumoto, Anne Marie Mazzola, Yusuf S. Nasrullah, Joseph Nneji, Huilan Ren, Athul Sanjeev, Madeleine Shay, Mary R. Stahley, Steven H.Y. Fan, Ying K. Tam, Nicole M. Gaudelli, Giuseppe Ciaramella, Leslie E. Stolz, Padma Malyala, Christopher J. Cheng, Kallanthottathil G. Rajeev, Ellen Rohde, Andrew M. Bellinger, Sekar Kathiresan

2021Nature768 citationsDOI

Topics & Concepts

CRISPRPCSK9Gene knockdownGenome editingIn vivoBiologyGenetic enhancementCholesterolGeneCancer researchComputational biologyBioinformaticsGeneticsLDL receptorLipoproteinEndocrinologyCRISPR and Genetic EngineeringMosquito-borne diseases and controlViral Infections and Immunology Research
In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates | Litcius