ArticleGene therapy2017
Site-directed RNA editing by adenosine deaminase acting on RNA for correction of the genetic code in gene therapy.
Article in Gene therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
27 citing papers in PubMed, 53 citations in OpenAlex.
- Dense RNA motif modifications enable robust in vivo prime editing and enhance efficiencies of diverse editing systems.Nature biomedical engineering · 2026Article
- A transcriptome-wide systematic search does not detect A-to-I RNA editing inGenome research · 2026Article
- ADAR-GPT: A continually fine-tuned language model for predicting A-to-I RNA editing sites.Proceedings of the National Academy of Sciences of the United States of America · 2026Article
- Single-strand deaminase-assisted editing for functional RNA manipulation.Nature biotechnology · 2026Article
- An ADAR2-mimic base editor for efficient C-to-U RNA editing in vivo.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Harnessing RNA base editing for diverse applications in RNA biology and RNA therapeutics.Advanced biotechnology · 2025Review
- Enhancing RNA editing efficiency and specificity with engineered ADAR2 guide RNAs.Molecular therapy. Nucleic acids · 2025Article
- RNA editing in disease: mechanisms and therapeutic potential.RNA (New York, N.Y.) · 2025Review
- Restoration of Genetic Code in Macular Mouse Fibroblasts via APOBEC1-Mediated RNA Editing.Biomolecules · 2025Article
- Programmable RNA base editing via targeted modifications.Nature chemical biology · 2024Review
- Detection of Developmental Asexual Stage-Specific RNA Editing Events inMicroorganisms · 2024Article
- Examination of Factors Affecting Site-Directed RNA Editing by the MS2-ADAR1 Deaminase System.Genes · 2023Article
- RNA Editing Therapeutics: Advances, Challenges and Perspectives on Combating Heart Disease.Cardiovascular drugs and therapy · 2023Review
- Site-directed A → I RNA editing as a therapeutic tool: moving beyond genetic mutations.RNA (New York, N.Y.) · 2023Article
- Identification of exceptionally potent adenosine deaminases RNA editors from high body temperature organisms.PLoS genetics · 2023Article
- Site-directed RNA editing by harnessing ADARs: advances and challenges.Functional & integrative genomics · 2022Review
- Targeted RNA editing: novel tools to study post-transcriptional regulation.Molecular cell · 2022Review
- Development of Therapeutic RNA Manipulation for Muscular Dystrophy.Frontiers in genome editing · 2022Review
- Site-directed RNA editing: recent advances and open challenges.RNA biology · 2021Review
- Rational Design of RNA Editing Guide Strands: Cytidine Analogs at the Orphan Position.Journal of the American Chemical Society · 2021Article
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Site-directed RNA editing is an important technique for correcting gene sequences and ultimately tuning protein function. In this study, we engineered the deaminase domain of adenosine deaminase acting on RNA (ADAR1) and the MS2 system to target-specific adenosines, with the goal of correcting G-to-A mutations at the RNA level. For this purpose, the ADAR1 deaminase domain was fused downstream of the RNA-binding protein MS2, which has affinity for the MS2 RNA. To direct editing to specific targets, we designed guide RNAs complementary to target RNAs. The guide RNAs directed the ADAR1 deaminase to the desired editing site, where it converted adenosine to inosine. To provide proof of principle, we used an allele of enhanced green fluorescent protein (EGFP) bearing a mutation at the 58th amino acid (TGG), encoding Trp, into an amber (TAG) or ochre (TAA) stop codon. In HEK-293 cells, our system could convert stop codons to read-through codons, thereby turning on fluorescence. We confirmed the specificity of editing at the DNA level by restriction fragment length polymorphism analysis and sequencing, and at the protein level by western blotting. The editing efficiency of this enzyme system was ~5%. We believe that this system could be used to treat genetic diseases resulting from G-to-A point mutations.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.