Genomic DNA at prime editing guide RNA-specified locus (pegRNA-specified locus)

Genomic DNA at a pegRNA-specified locus refers to the precise chromosomal site targeted for modification by prime editing technology. Prime editing is a versatile genome-editing method that employs a fusion protein comprising a catalytically impaired Cas9 nickase and a reverse transcriptase, directed by a prime editing guide RNA (pegRNA) (Anzalone et al., 2019). The pegRNA serves a dual purpose: it identifies the target DNA sequence and provides the template for the desired genetic change, allowing for insertions, deletions, and all twelve possible base-to-base substitutions (Chen & Liu, 2023). This target is central to the treatment of various genetic diseases by enabling the direct correction of pathogenic mutations within the patient's genome. Unlike traditional CRISPR-Cas9, which relies on double-strand breaks, prime editing nicks only one strand, potentially reducing the risk of uncontrolled indels and chromosomal rearrangements. Therapeutic applications targeting specific loci are currently under investigation for conditions such as sickle cell disease and chronic granulomatous disease, with candidates like PM-359 entering clinical evaluation (Prime Medicine, 2024).

Other names

Prime editing target sitepegRNA target sequenceGenomic target of prime editorPrime editing locus

Prime editing (search-and-replace genome editing) involving reverse transcription of an RNA template into a nicked genomic DNA strand