fig4

Gene therapy for inborn errors of immunity: past progress, current status and future directions

Figure 4. Schematic diagram showing the mechanism of adenine base editing. A gRNA targets a Cas nickase tethered to an adenine/cytosine deaminase to a specific sequence within the genome. There, specific deamination of target bases within a base editing window takes place, resulting in A:T - > G:C or C:G - > T:A transition mutations. Created in BioRender. Orf K (2025) https://BioRender.com/udxwt5j. ABEs: Adenine base editors; gRNA: guide RNA.

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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