In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA
Abstract
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...
Paper Details
Title
In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA
DOI
Published Date
Jan 25, 2021
Journal
Volume
5
Issue
2
Pages
179 - 189
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Notes
History