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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
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

Internet

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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