In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA

Base editors are RNA-programmable deaminases enabling precise single-base conversions on 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 modificat...

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Udgivet i:Nat Biomed Eng
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: 2021
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7610981/
https://ncbi.nlm.nih.gov/pubmed/33495639
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41551-020-00671-z
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