Ontology highlight
ABSTRACT:
SUBMITTER: Song CQ
PROVIDER: S-EPMC6986236 | biostudies-literature | 2020 Jan
REPOSITORIES: biostudies-literature
Song Chun-Qing CQ Jiang Tingting T Richter Michelle M Rhym Luke H LH Koblan Luke W LW Zafra Maria Paz MP Schatoff Emma M EM Doman Jordan L JL Cao Yueying Y Dow Lukas E LE Zhu Lihua Julie LJ Anderson Daniel G DG Liu David R DR Yin Hao H Xue Wen W
Nature biomedical engineering 20190225 1
In contrast to traditional CRISPR-Cas9 homology-directed repair, base editing can correct point mutations without supplying a DNA-repair template. Here we show in a mouse model of tyrosinaemia that hydrodynamic tail-vein injection of plasmid DNA encoding the adenine base editor (ABE) and a single-guide RNA (sgRNA) can correct an A>G splice-site mutation. ABE treatment partially restored splicing, generated fumarylacetoacetate hydrolase (FAH)-positive hepatocytes in the liver, and rescued weight ...[more]