Target intelligence / Profile preview

DNA base editor (CRISPR-guided deaminase fusion protein) (Base editor (BE); specific versions include cytosine base editor (CBE) and adenine base editor (ABE))

Target
Base editor (BE); specific versions include cytosine base editor (CBE) and adenine base editor (ABE)
Molecular classification
Other (engineered fusion protein/gene editing tool), Enzyme (contains deaminase activity), DNA modifying enzyme, Targeted genome editing tool
01

Overview

DNA base editors are engineered fusion proteins combining a catalytically impaired CRISPR-associated nuclease (commonly Cas9) with a single-stranded DNA deaminase enzyme, capable of introducing precise, programmable nucleotide substitutions at specific genomic loci without creating double-stranded DNA breaks[1][3][5][7]. The system uses a guide RNA to direct the fusion protein to the target DNA sequence, where the deaminase catalyzes conversion of cytosine to uracil (in cytosine base editors) or adenine to inosine (in adenine base editors); these modifications are recognized and fixed as C→T or A→G transitions by cellular repair pathways[3][5][7]. Such tools are research and medical candidates for correcting pathogenic point mutations in living cells and tissues, with advantages in efficiency and precision but significant ongoing concerns regarding specificity and safety.

Other names
CRISPR base editorCytosine base editor (CBE)Adenine base editor (ABE)Cas9-deaminase fusion proteinCRISPR-guided base editor
02

Mechanism of action

Induces targeted chemical conversion of a single nucleotide (C→T or A→G) in genomic DNA through RNA-guided binding and enzymatic deamination[3][5][7]

03

Biological functions

DNA sequence editingSingle-nucleotide modificationPoint mutation correction or induction
04

Disease associations

Cancer (correction or introduction of oncogenic point mutations)Monogenic genetic diseases (sickle cell disease, cystic fibrosis, etc.)Other genetic disorders involving point mutations
05

Safety considerations

Off-target DNA editingBystander editing (modification of adjacent bases)Unintended immunogenicityPersistent or heritable genomic changes in germlineUnpredictable long-term consequences of genome edits[5][6][7]
06

Interacting drugs

None in standard pharmacopeia; under research for delivery methods (e.g., viral vectors, nanoparticles); theoretical inhibitors could be developed for off-switches
07

Biomarkers

Edited DNA sequence at targeted locus (verification via sequencing)Absence or presence of specific point mutation

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