Base editing: advances and therapeutic opportunities

EM Porto, AC Komor, IM Slaymaker… - Nature Reviews Drug …, 2020 - nature.com
Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

Base editing: precision chemistry on the genome and transcriptome of living cells

HA Rees, DR Liu - Nature reviews genetics, 2018 - nature.com
RNA-guided programmable nucleases from CRISPR systems generate precise breaks in
DNA or RNA at specified positions. In cells, this activity can lead to changes in DNA …

A cytosine deaminase for programmable single-base RNA editing

OO Abudayyeh, JS Gootenberg, B Franklin, J Koob… - Science, 2019 - science.org
Programmable RNA editing enables reversible recoding of RNA information for research
and disease treatment. Previously, we developed a programmable adenosine-to-inosine (A …

RNA editing with CRISPR-Cas13

DBT Cox, JS Gootenberg, OO Abudayyeh, B Franklin… - Science, 2017 - science.org
Nucleic acid editing holds promise for treating genetic disease, particularly at the RNA level,
where disease-relevant sequences can be rescued to yield functional protein products. Type …

Efficient in vitro and in vivo RNA editing via recruitment of endogenous ADARs using circular guide RNAs

D Katrekar, J Yen, Y Xiang, A Saha, D Meluzzi… - Nature …, 2022 - nature.com
Recruiting endogenous adenosine deaminases using exogenous guide RNAs to edit
cellular RNAs is a promising therapeutic strategy, but editing efficiency and durability remain …

CRISPR technologies for genome, epigenome and transcriptome editing

L Villiger, J Joung, L Koblan, J Weissman… - … Reviews Molecular Cell …, 2024 - nature.com
Our ability to edit genomes lags behind our capacity to sequence them, but the growing
understanding of CRISPR biology and its application to genome, epigenome and …

Engineered circular ADAR-recruiting RNAs increase the efficiency and fidelity of RNA editing in vitro and in vivo

Z Yi, L Qu, H Tang, Z Liu, Y Liu, F Tian, C Wang… - Nature …, 2022 - nature.com
Current methods for programmed RNA editing using endogenous ADAR enzymes and
engineered ADAR-recruiting RNAs (arRNAs) suffer from low efficiency and bystander off …

Programmable eukaryotic protein synthesis with RNA sensors by harnessing ADAR

K Jiang, J Koob, XD Chen, RN Krajeski, Y Zhang… - Nature …, 2023 - nature.com
Programmable approaches to sense and respond to the presence of specific RNAs in
biological systems have broad applications in research, diagnostics, and therapeutics. Here …

A-to-I RNA editing—immune protector and transcriptome diversifier

E Eisenberg, EY Levanon - Nature Reviews Genetics, 2018 - nature.com
Modifications of RNA affect its function and stability. RNA editing is unique among these
modifications because it not only alters the cellular fate of RNA molecules but also alters …

Precision RNA base editing with engineered and endogenous effectors

LS Pfeiffer, T Stafforst - Nature Biotechnology, 2023 - nature.com
RNA base editing refers to the rewriting of genetic information within an intact RNA molecule
and serves various functions, such as evasion of the endogenous immune system and …