Publication: In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA
In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA
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Villiger, L., Rothgangl, T., Witzigmann, D., Oka, R., Lin, P. J. C., Qi, W., Janjuha, S., Berk, C., Ringnalda, F., Beattie, M. B., Stoffel, M., Thöny, B., Hall, J., Rehrauer, H., van Boxtel, R., Tam, Y. K., & Schwank, G. (2021). In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA. Nature Biomedical Engineering, 5(2), 179–189. https://doi.org/10.1038/s41551-020-00671-z
Abstract
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Base editors are RNA-programmable deaminases that enable precise single-base conversions in genomic DNA. However, off-target activity is a concern in the potential use of base editors to treat genetic diseases. Here, we report unbiased analyses of transcriptome-wide and genome-wide off-target modifications effected by cytidine base editors in the liver of mice with phenylketonuria. The intravenous delivery of intein-split cytidine base editors by dual adeno-associated viruses led to the repair of the disease-causing mutation without g
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- Villiger, Lukas
- Rothgangl, Tanja
- Witzigmann, Dominik
- Oka, Rurika
- Lin, Paulo J C
- Qi, Weihong
- Janjuha, Sharan
- Berk, Christian
- Ringnalda, Femke
- Beattie, Mitchell B
- Stoffel, Markus
- Thöny, Beat
- Hall, Jonathan
- Rehrauer, Hubert
- van Boxtel, Ruben
- Tam, Ying K
- Schwank, Gerald
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Villiger, L., Rothgangl, T., Witzigmann, D., Oka, R., Lin, P. J. C., Qi, W., Janjuha, S., Berk, C., Ringnalda, F., Beattie, M. B., Stoffel, M., Thöny, B., Hall, J., Rehrauer, H., van Boxtel, R., Tam, Y. K., & Schwank, G. (2021). In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA. Nature Biomedical Engineering, 5(2), 179–189. https://doi.org/10.1038/s41551-020-00671-z
