Treatment of a metabolic liver disease by in vivo prime editing in mice
Abstract
Prime editing is a highly versatile CRISPR-based genome editing technology with the potential to correct the vast majority of pathogenic mutations (1) . However, correction of a disease phenotype in vivo in somatic tissues has not been demonstrated thus far. Here, we establish proof-of-concept for in vivo prime editing and repair the metabolic liver disease phenylketonuria (PKU) in mice. We first developed a size-reduced Sp Cas9 prime editor...
Paper Details
Title
Treatment of a metabolic liver disease by in vivo prime editing in mice
DOI
Published Date
Aug 17, 2021
Journal
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Notes
History