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ecRESCUE: a novel ecDHFR-regulated RESCUE system with reduced RNA off-targeting activity

التفاصيل البيبلوغرافية
العنوان: ecRESCUE: a novel ecDHFR-regulated RESCUE system with reduced RNA off-targeting activity
المؤلفون: Yihan Wang, Guo Li, Xiangyang Li, Yuzhe Wang, Xingxu Huang, Xiaoxiang Hu, Jianen Gao
المصدر: Cell Communication and Signaling, Vol 19, Iss 1, Pp 1-9 (2021)
بيانات النشر: BMC, 2021.
سنة النشر: 2021
المجموعة: LCC:Medicine
LCC:Cytology
مصطلحات موضوعية: Dihydrofolate reductase destabilization domain, ecRESCUE, Off-targeting activity, RESCUE system, RNA base editing, Medicine, Cytology, QH573-671
الوصف: Abstract The currently available RESCUE RNA base editing system demonstrates considerable potential for the treatment of genetic diseases at the transcriptional level. However, the relatively high incidence of off-target events hampers the precise RNA editing, thereby limiting its use in the clinical setting. This study describes a new RNA base editing method, named ecRESCUE, which utilizes inducible stabilization of the protein ecDHFR DD fused at the C-terminal of the original RESCUE system. In vitro experiments in 293T cells showed that the ecRESCUE editor markedly reduced the incidence of off-target single nucleotide polymorphisms without affecting the RNA A-to-I and C-to-U base editing efficiency. Altogether, these results demonstrate that the inducible ecRESCUE system represents an attractive approach to regulate and improve the outcome of the available RNA base editor with reduced off-targeting activity. Video Abstract
نوع الوثيقة: article
وصف الملف: electronic resource
اللغة: English
تدمد: 1478-811X
Relation: https://doaj.org/toc/1478-811X
DOI: 10.1186/s12964-021-00759-2
URL الوصول: https://doaj.org/article/5dcf0896f382412fa00abba7917c7be6
رقم الأكسشن: edsdoj.5dcf0896f382412fa00abba7917c7be6
قاعدة البيانات: Directory of Open Access Journals
الوصف
تدمد:1478811X
DOI:10.1186/s12964-021-00759-2