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 …

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 …

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 …

Recruiting endogenous adenosine deaminases using exogenous guide RNAs to edit
cellular RNAs is a promising therapeutic strategy, but editing efficiency and durability remain …

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 …

Inherited retinal diseases (IRDs) are a clinically complex and heterogenous group of visual
impairment phenotypes caused by pathogenic variants in at least 277 nuclear and …

Programmable approaches to sense and respond to the presence of specific RNAs in
biological systems have broad applications in research, diagnostics, and therapeutics. Here …

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 …

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 …

Summary Nonsense mutations, accounting for> 20% of disease-associated mutations, lead
to premature translation termination. Replacing uridine with pseudouridine in stop codons …