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Utilizing AAV-mediated LEAPER 2.0 for programmable RNA editing in non-human primates and nonsense mutation correction in humanized Hurler syndrome mice
BACKGROUND: The endogenous adenosine deaminases acting on RNA (ADAR) have been harnessed to facilitate precise adenosine-to-inosine editing on RNAs. However, the practicability of this approach for therapeutic purposes is still ambiguous due to the variable expression of intrinsic ADAR across variou...
Autores principales: | Yi, Zongyi, Zhao, Yanxia, Yi, Zexuan, Zhang, Yongjian, Tang, Gangbin, Zhang, Xiaoxue, Tang, Huixian, Zhang, Wei, Zhao, Ying, Xu, Huayuan, Nie, Yuyang, Sun, Xueqing, Xing, Lijun, Dai, Lian, Yuan, Pengfei, Wei, Wensheng |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10591355/ https://www.ncbi.nlm.nih.gov/pubmed/37872590 http://dx.doi.org/10.1186/s13059-023-03086-6 |
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