Health AI Central
Peer-Reviewed Publication
Nature2026September 9, 2026Journal Article

Highly efficient base editing at PCSK9 and normal human embryo development.

Stepan Jerabek1,2, Chanju Jung3, Michelle Kappy4, Qiaojin Zhao1, Julie Sung1, Ning Wang1, Euihyun Kim1, Jimin Kim1, Marcos Iuri Roos Kulmann5,6, Madeleine Bliss King7,8, Mitchell John McAndrew8, Meng Li9, Sakshi Bhatele1, Melisa Isado1, Hong-Su Jang10,11, Michal Dolezal2, Robert Prosser4, Shuangyi Xu1, Gue-Ho Hwang12, Iva Pichova2, Jia Xu13, Diego Marin13,14, Jae-Sung Woo11, Sangsu Bae3,12,15, Nathan Treff13,16,17, Audrone Lapinaite8,18,19, Dieter Egli20,21,22
1Columbia University, Department of Pediatrics, Division of Molecular Genetics, New York,, NY, USA.
2Institute of Organic Chemistry and Biochemistry of the Czech Academy of Sciences, Prague 6, Czech Republic.
3Department of Biomedical Sciences, Seoul National University College of Medicine, Seoul, Republic of Korea.
4Division of Reproductive Endocrinology and Infertility, Department of Obstetrics and Gynecology, Columbia University, New York,, NY, USA.
5Institute of Molecular Genetics of the Czech Academy of Sciences, Prague 4, Czech Republic.
6Department of Cell Biology, Charles University, Prague 2, Czech Republic.
7School of Molecular Sciences, Arizona State University, Tempe, AZ, USA.
8Gavin Herbert Eye Institute, Brunson Center for Translational Vision Research, Department of Ophthalmology & Visual Sciences, University of California Irvine, School of Medicine, Irvine, CA, USA.
9Department of Medicine, Columbia University, New York,, NY, USA.
10Department of Life Sciences, Korea University, Seoul, Republic of Korea.
11Center for Biomolecular and Cellular Structure, Institute for Basic Science, Daejeon, Republic of Korea.
12Medical Research Center of Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, Republic of Korea.
13Genomic Prediction, Inc., Hackettstown, NJ, USA.
14Department of Genetics, Rutgers University, Piscataway, NJ, USA.
15Cancer Research Institute, Seoul National University College of Medicine, Seoul, Republic of Korea.
16Department of Obstetrics, Gynecology, and Reproductive Sciences, Rutgers University, New Brunswick, NJ, USA.
17Nucleus Genomics, Inc., New York, NY, USA.
18Department of Biomedical Engineering, University of California Irvine, Samueli School of Engineering, Irvine, CA, USA.
19Department of Biological Chemistry, University of California Irvine, School of Medicine, Irvine, CA, USA.
20Columbia University, Department of Pediatrics, Division of Molecular Genetics, New York,, NY, USA. de2220@cumc.columbia.edu.
21Division of Reproductive Endocrinology and Infertility, Department of Obstetrics and Gynecology, Columbia University, New York,, NY, USA. de2220@cumc.columbia.edu.
22Columbia Stem Cell Initiative, Columbia University, New York, NY, USA. de2220@cumc.columbia.edu.
PubMed DOI

Abstract

Cas9-based tools enable programmable DNA lesions for studying repair outcomes, gene function, and genome correction. In human embryos, Cas9-induced DNA double-strand breaks are genotoxic, causing frequent aneuploidy and large deletions1,2. Here, we evaluate DNA repair outcomes at nicks and mismatches introduced by base editors at the PCSK9 and HBG loci in human embryos. Delivering ABE8e-V106W as a…

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