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RNA editing

Molecular classification
Other
01

Overview

RNA editing is a post-transcriptional molecular process in which specific nucleotides within an RNA molecule are altered, inserted, or deleted after transcription, resulting in an RNA sequence that can differ from its corresponding genomic DNA. This can include base substitutions (e.g., adenosine-to-inosine [A-to-I] and cytidine-to-uridine [C-to-U] deaminations), as well as insertions or deletions of nucleotides, often mediated by guide RNAs or specific deaminase enzymes. RNA editing occurs in various types of RNA (mRNA, tRNA, rRNA, miRNA) and in multiple cellular locations including the nucleus, cytoplasm, mitochondria, and plastids[1][3][6][7]. It provides a regulatory layer that diversifies the transcriptome and proteome, enables cellular adaptation, and is implicated in the pathogenesis of diseases such as cancer, neurodegeneration, and cardiovascular disorders[4][6]. While not itself a single molecular target, RNA editing is a diverse family of processes and is being explored as a therapeutic avenue for precise, reversible correction of pathogenic RNA sequences; these approaches remain experimental and do not yet involve well-defined drug–target relationships[2].

Other names
RNA modification
02

Biological functions

Regulation of gene expressionGeneration of protein diversityRNA stabilityTranslational control
03

Disease associations

CancerNeurodegenerative disease (e.g. ALS)Cardiovascular disease (via modified lipoprotein function)Other
04

Safety considerations

Potential for off-target effects if engineered for therapyTransient effects (challenges for durable therapies)Reversibility may limit long-term benefit

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