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DNA double-strand break at user-specified genomic locus via guide RNA-directed cleavage
- Molecular classification
- Other (this refers to a molecular event, not a discrete molecule or receptor)
01
Overview
DNA double-strand break at a user-specified genomic locus via guide RNA-directed cleavage refers to the specific, targeted cutting of double-stranded DNA directed by an engineered guide RNA system, most commonly implemented using CRISPR/Cas9 technology. In this method, a Cas9 nuclease is directed to a user-defined DNA sequence by a complementary guide RNA, cutting both DNA strands a few nucleotides upstream of a PAM sequence. The resulting double-strand break is repaired by endogenous cellular pathways—either non-homologous end joining (NHEJ), which is error-prone and can create insertions or deletions, or homology-directed repair (HDR), which can enable precise DNA insertion or correction if a template is supplied[2][3][4][5]. This event is foundational to genome editing but is not itself a target molecule or receptor; rather, it is a mechanistic event engineered for varied biological applications.
- Other names
- CRISPR/Cas9-induced DNA double-strand breakRNA-guided DNA cleavageGenome editing cleavage site
02
Biological functions
Genome editingDNA recombinationDNA repair pathway activation (including non-homologous end joining and homologous recombination)[2][3][4][5]
03
Disease associations
Other (the break itself is not disease-related, but the editing tool is used in various disease models and gene therapies)
04
Safety considerations
Off-target cutting (CRISPR/Cas9 and other genome editors may induce unwanted DNA breaks at unintended loci)[4]Genomic instability (erroneous repair can lead to insertions, deletions, translocations, or mutations)[2][3][4]Unintended consequences (including possible oncogenic rearrangements or cell death)[2][4]
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