Publication: In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
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Rothgangl, T., Dennis, M. K., Lin, P. J. C., Oka, R., Witzigmann, D., Villiger, L., Qi, W., Hruzova, M., Kissling, L., Lenggenhager, D., Borrelli, C., Egli, S., Frey, N., Bakker, N., Walker, J. A., Kadina, A. P., Victorov, D. V., Pacesa, M., Kreutzer, S., … Schwank, G. (2021). In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels. Nature Biotechnology, 39(8), 949–957. https://doi.org/10.1038/s41587-021-00933-4
Abstract
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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 mac
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Creators (Authors)
- Rothgangl, Tanja
- Dennis, Melissa K
- Lin, Paulo J C
- Oka, Rurika
- Witzigmann, Dominik
- Villiger, Lukas
- Qi, Weihong
- Hruzova, Martina
- Kissling, Lucas
- Lenggenhager, Daniela
- Borrelli, Costanza
- Egli, Sabina
- Frey, Nina
- Bakker, Noëlle
- Walker, John A
- Kadina, Anastasia P
- Victorov, Denis V
- Pacesa, Martin
- Kreutzer, Susanne
- Kontarakis, Zacharias
- Moor, Andreas
- Jinek, Martin
- Weissman, Drew
- Stoffel, Markus
- van Boxtel, Ruben
- Holden, Kevin
- Pardi, Norbert
- Thöny, Beat
- Häberle, Johannes
- Tam, Ying K
- Semple, Sean C
- Schwank, Gerald
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Publications of Department of Biochemistry
Publications of Functional Genomics Center Zurich
Publications of Institute of Pathology and Molecular Pathology
Publications of Institute of Pharmacology and Toxicology
Publications of Institute of Pharmacology and Toxicology
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Rothgangl, T., Dennis, M. K., Lin, P. J. C., Oka, R., Witzigmann, D., Villiger, L., Qi, W., Hruzova, M., Kissling, L., Lenggenhager, D., Borrelli, C., Egli, S., Frey, N., Bakker, N., Walker, J. A., Kadina, A. P., Victorov, D. V., Pacesa, M., Kreutzer, S., … Schwank, G. (2021). In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels. Nature Biotechnology, 39(8), 949–957. https://doi.org/10.1038/s41587-021-00933-4
