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Results in Journal Nature Biotechnology: 17,032

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Nature Biotechnology, Volume 39, pp 653-653; doi:10.1038/s41587-021-00957-w

Cormac Sheridan
Nature Biotechnology, Volume 39, pp 650-652; doi:10.1038/s41587-021-00955-y

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Nature Biotechnology, Volume 39, pp 652-652; doi:10.1038/s41587-021-00963-y

Nature Biotechnology, Volume 39, pp 780-780; doi:10.1038/s41587-021-00956-x

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Nature Biotechnology, Volume 39, pp 685-685; doi:10.1038/s41587-021-00952-1

Nature Biotechnology, Volume 39, pp 684-684; doi:10.1038/s41587-021-00958-9

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Abasi Ene Abong, Katrine Bosley, Charlotte Casebourn, Priscilla Chan, Janice Chen, Michael Chen, George Church, John Cumbers, Tomas de Wouters, Heather Dewey-Hagborg, et al.
Nature Biotechnology, Volume 39, pp 654-660; doi:10.1038/s41587-021-00941-4

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Nature Biotechnology, Volume 39, pp 649-649; doi:10.1038/s41587-021-00961-0

Elie Dolgin
Nature Biotechnology, Volume 39, pp 647-649; doi:10.1038/s41587-021-00954-z

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Nature Biotechnology, Volume 39, pp 685-685; doi:10.1038/s41587-021-00953-0

Nature Biotechnology, Volume 39, pp 650-650; doi:10.1038/s41587-021-00964-x

Anoop Manjunath, Hongyu Li, Shuchen Song, Zhixing Zhang, Shu Liu, Nathan Kahrobai, Arya Gowda, Angelina Seffens, ,
Nature Biotechnology, Volume 39, pp 678-683; doi:10.1038/s41587-021-00940-5

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, Marina Kvaskoff,
Nature Biotechnology, Volume 39, pp 776-779; doi:10.1038/s41587-021-00951-2

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Ken Garber
Nature Biotechnology; doi:10.1038/d41587-021-00006-6

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, Nina Frahm, David Winickoff, David Benrimoh, , Gary Marchant
Nature Biotechnology, Volume 39, pp 661-664; doi:10.1038/s41587-021-00947-y

Nature Biotechnology, Volume 39, pp 671-677; doi:10.1038/s41587-021-00945-0

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Nature Biotechnology, Volume 39, pp 645-645; doi:10.1038/s41587-021-00967-8

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Eleni P. Mimitou, , Kelvin Y. Chen, Andre L. Zorzetto-Fernandes, Yuhan Hao, Yusuke Takeshima, Wendy Luo, Tse-Shun Huang, Bertrand Z. Yeung, Efthymia Papalexi, et al.
Nature Biotechnology pp 1-13; doi:10.1038/s41587-021-00927-2

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Edward Zhao, Matthew R. Stone, Xing Ren, Jamie Guenthoer, Kimberly S. Smythe, Thomas Pulliam, Stephen R. Williams, Cedric R. Uytingco, Sarah E. B. Taylor, , et al.
Nature Biotechnology pp 1-10; doi:10.1038/s41587-021-00935-2

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Ravian L. van Ineveld, , Maria Alieva, Sam de Blank, Mario Barrera Roman, Esmée J. van Vliet, Clara Martínez Mir, Hannah R. Johnson, Frank L. Bos, , et al.
Nature Biotechnology pp 1-7; doi:10.1038/s41587-021-00926-3

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Esther Landhuis
Nature Biotechnology; doi:10.1038/d41587-021-00004-8

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Chi-Yun Wu, Billy T. Lau, Heon Seok Kim, , Susan M. Grimes, ,
Published: 20 May 2021
Nature Biotechnology pp 1-11; doi:10.1038/s41587-021-00911-w

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, Santoshi Biswanath, Clara-Milena Farr, Victoria Lupanow, Jana Teske, Katharina Ritzenhoff, Annika Franke, , Emiliano Bolesani, , et al.
Nature Biotechnology, Volume 39, pp 775-775; doi:10.1038/s41587-021-00960-1

Tanja Rothgangl, Melissa K. Dennis, Paulo J. C. Lin, Rurika Oka, , Lukas Villiger, Weihong Qi, Martina Hruzova, Lucas Kissling, Daniela Lenggenhager, et al.
Nature Biotechnology pp 1-9; doi:10.1038/s41587-021-00933-4

Abstract:
Most known pathogenic point mutations in humans are C•G to T•A substitutions, which can be directly repaired by adenine base editors (ABEs). In this study, we investigated the efficacy and safety of ABEs in the livers of mice and cynomolgus macaques for the reduction of blood low-density lipoprotein (LDL) levels. Lipid nanoparticle–based delivery of mRNA encoding an ABE and a single-guide RNA targeting PCSK9, a negative regulator of LDL, induced up to 67% editing (on average, 61%) in mice and up to 34% editing (on average, 26%) in macaques. Plasma PCSK9 and LDL levels were stably reduced by 95% and 58% in mice and by 32% and 14% in macaques, respectively. ABE mRNA was cleared rapidly, and no off-target mutations in genomic DNA were found. Re-dosing in macaques did not increase editing, possibly owing to the detected humoral immune response to ABE upon treatment. These findings support further investigation of ABEs to treat patients with monogenic liver diseases.
James W. Weis, Joseph M. Jacobson
Published: 17 May 2021
Nature Biotechnology pp 1-8; doi:10.1038/s41587-021-00907-6

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Karl Petri, Weiting Zhang, Junyan Ma, Andrea Schmidts, Hyunho Lee, Joy E. Horng, Daniel Y. Kim, Ibrahim C. Kurt, , , et al.
Nature Biotechnology pp 1-1; doi:10.1038/s41587-021-00939-y

Oguzhan Begik, , Leszek P. Pryszcz, Jose Miguel Ramirez, Rebeca Medina, Ivan Milenkovic, Sonia Cruciani, , Helaine Graziele Santos Vieira, Aldema Sas-Chen, et al.
Nature Biotechnology pp 1-14; doi:10.1038/s41587-021-00915-6

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Michael Francisco
Nature Biotechnology, Volume 39, pp 643-643; doi:10.1038/s41587-021-00919-2

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Laura DeFrancesco
Nature Biotechnology, Volume 39, pp 538-539; doi:10.1038/s41587-021-00918-3

Nature Biotechnology, Volume 39, pp 533-533; doi:10.1038/s41587-021-00931-6

Nature Biotechnology, Volume 39, pp 644-644; doi:10.1038/s41587-021-00923-6

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Laura DeFrancesco
Nature Biotechnology, Volume 39, pp 536-537; doi:10.1038/s41587-021-00917-4

, Burcu Kilic
Nature Biotechnology, Volume 39, pp 546-548; doi:10.1038/s41587-021-00912-9

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Nature Biotechnology, Volume 39, pp 535-535; doi:10.1038/s41587-021-00921-8

Cormac Sheridan
Nature Biotechnology, Volume 39, pp 533-534; doi:10.1038/s41587-021-00920-9

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Nature Biotechnology, Volume 39, pp 531-531; doi:10.1038/s41587-021-00929-0

Nature Biotechnology, Volume 39, pp 550-550; doi:10.1038/s41587-021-00914-7

Michael Francisco
Nature Biotechnology, Volume 39, pp 532-532; doi:10.1038/s41587-021-00932-5

Nature Biotechnology, Volume 39, pp 534-534; doi:10.1038/s41587-021-00930-7

Nature Biotechnology, Volume 39, pp 549-549; doi:10.1038/s41587-021-00924-5

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Sarah J. Shareef, Samantha M. Bevill, Ayush T. Raman, , , ,
Nature Biotechnology pp 1-9; doi:10.1038/s41587-021-00910-x

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Nature Biotechnology pp 1-2; doi:10.1038/s41587-021-00916-5

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Nature Biotechnology, Volume 39, pp 527-527; doi:10.1038/s41587-021-00937-0

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, Simon Little, Randy Perrone, Robert Wilt, Coralie de Hemptinne, Maria S. Yaroshinsky, Caroline A. Racine, Sarah S. Wang, Jill L. Ostrem, Paul S. Larson, et al.
Nature Biotechnology pp 1-8; doi:10.1038/s41587-021-00897-5

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Thamotharampillai Dileepan, , , , Peter D. Krueger, Brian D. Evavold,
Nature Biotechnology pp 1-6; doi:10.1038/s41587-021-00893-9

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Timothy J. Cary, , Long Zhang, Ryan M. Routsong, Antonio J. Palazzo, Stuart E. Strand,
Nature Biotechnology pp 1-4; doi:10.1038/s41587-021-00909-4

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, Christopher Douville, Jonathan C. Dudley, Brian J. Mog, Maria Popoli, Janine Ptak, Lisa Dobbyn, Natalie Silliman, Joy Schaefer, Jeanne Tie, et al.
Nature Biotechnology pp 1-8; doi:10.1038/s41587-021-00900-z

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