Making base editing accessible: Evaluating computational workflows for ABE-mediated gene knockout

Since the development of the first base editors, continued evolution has yielded a selection of editors that have been proved to be highly effective and precise in research and their first clinical use.1 Built on CRISPR technology, base editors utilize modified Cas proteins fused to a deaminase domain and directed by a single-guide RNA (sgRNA) to a specific genomic locus. Within a defined editing window, the deaminase catalyzes the conversion of one nucleotide to another without generating double-strand breaks (DSBs) (Figure 1).

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Publication Details

Journal
Molecular Therapy — Nucleic Acids
Published
2026-07-27
DOI
https://doi.org/10.1016/j.omtn.2026.103014
Primary Topic
CRISPR and Genetic Engineering
Type
article
Field-Weighted Citation Impact
0.00

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article

Making base editing accessible: Evaluating computational workflows for ABE-mediated gene knockout

J. Jackow, Aidin Kazemizadeh, James Williamson
Molecular Therapy — Nucleic Acids
CRISPR and Genetic Engineering
article

Making base editing accessible: Evaluating computational workflows for ABE-mediated gene knockout

J. Jackow, Aidin Kazemizadeh, James Williamson
article en

Abstract

Since the development of the first base editors, continued evolution has yielded a selection of editors that have been proved to be highly effective and precise in research and their first clinical use.1 Built on CRISPR technology, base editors utilize modified Cas proteins fused to a deaminase domain and directed by a single-guide RNA (sgRNA) to a specific genomic locus. Within a defined editing window, the deaminase catalyzes the conversion of one nucleotide to another without generating double-strand breaks (DSBs) (Figure 1).

Molecular Therapy — Nucleic AcidsVol. 37(3)
King's College London (GB)
Epidermolysis Bullosa Medical Research Foundation, Dystrophic Epidermolysis Bullosa Research Association, Cure EB, British Skin Foundation, Rosetrees Trust
Openalex Percentile: Top 15%
CRISPR and Genetic Engineering
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