Amelioration of an Inherited Metabolic Liver Disease through Creation of a De Novo Start Codon by Cytidine Base Editing

Base editing technology efficiently generates nucleotide conversions without inducing excessive double-strand breaks (DSBs), which makes it a promising approach for genetic disease therapy. In this study, we generated a novel hereditary tyrosinemia type 1 (HT1) mouse model, which contains a start co...

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Veröffentlicht in:Mol Ther
Hauptverfasser: Yang, Lei, Wang, Liren, Huo, Yanan, Chen, Xi, Yin, Shuming, Hu, Yaqiang, Zhang, Xiaohui, Zheng, Rui, Geng, Hongquan, Han, Honghui, Ma, Xueyun, Liu, Meizhen, Li, Haibo, Yu, Weishi, Liu, Mingyao, Wang, Jun, Li, Dali
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2020
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7335753/
https://ncbi.nlm.nih.gov/pubmed/32413280
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.05.001
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