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RNA AND DNA BASE EDITING VIA ENGINEERED ADAR RECRUITMENT
RNA AND DNA BASE EDITING VIA ENGINEERED ADAR RECRUITMENT
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机译:通过工程化ADAR招聘RNA和DNA基础编辑
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摘要
Disclosed herein is a system to recruit ADARs to catalyze therapeutic editing of point mutations via the use of engineered RNA scaffolds, engineered DNA scaffolds or DNA-RNA hybrid scaffolds. The system comprises an engineered ADAR2 guide RNA (adRNA) that bears a 20-l00bp complementarity with the target RNA and ADAR2 recruiting domain from the GluR2 mRNA at either or both the 5' end or the 3' end.
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