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A Novel Engineered U7 Small Nuclear RNA Scaffold Greatly Increases in vitro and in vivo ADAR-Mediated Programmable RNA Base Editing

2024-10-01

Abstract excerpt

Custom RNA base editing using the endogenous human Adenosine Deaminase Acting on RNA (ADAR) enzyme presents a promising approach for precision therapeutics, alleviating concerns of permanent DNA damage or immunogenicity from 1 foreign bacterial proteins such as CRISPR/Cas. ADAR can be directed to act on therapeutic RNA targets by antisense guide RNAs (gRNAs) that create a substrate for ADAR’s adenosine-to-inosine...

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Literature Corpus work: 4050cd00-d876-5434-a508-4fb668bd00c3
DOI: 10.1101/2024.09.29.615721

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