Adenine base editing in an adult mouse model of tyrosinemia

Unlike 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 tyrosinemia that hydrodynamic tail-vein injection of plasmid DNA encoding the adenine base editor (ABE) and a single guide RNA...

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Pubblicato in:Nat Biomed Eng
Autori principali: Song, Chun-Qing, Jiang, Tingting, Richter, Michelle, Rhym, Luke H., Koblan, Luke W., Zafra, Maria Paz, Schatoff, Emma M, Doman, Jordan L., Cao, Yueying, Dow, Lukas E., Zhu, Lihua Julie, Anderson, Daniel G, Liu, David R., Yin, Hao, Xue, Wen
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2019
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6986236/
https://ncbi.nlm.nih.gov/pubmed/31740768
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41551-019-0357-8
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