In vivo base and prime editing for cancer
In vivo base and prime editing would rewrite a cancer's DNA letter by letter inside the body. It works in the liver for inherited disease; nobody has yet corrected a cancer this way in a person.
Base editors change a single DNA letter without cutting; prime editors write short new sequences. Both are in the clinic for inherited liver and blood disease, and base editing is used ex vivo to build allogeneic CAR-T cells. Direct in vivo correction of a cancer driver, restoring TP53 or disabling a mutant KRAS allele, had not entered human trials by September 2026: solid-tumour delivery, editing every malignant cell, and the fact that a corrected cell must still out-compete an uncorrected one are all unsolved.
How it works
A catalytically impaired Cas protein fused to a deaminase or reverse transcriptase is guided to a locus and edits it without a double-strand break; delivered by lipid nanoparticle or virus.
- Acts on the causal lesion, not a downstream protein
- No double-strand breaks, so fewer translocations than nuclease editing
- Re-programmable by changing the guide
- No in vivo oncology trial yet (2026)
- Delivery reaches liver far better than solid tumours
- Editing a fraction of cells may not change tumour behaviour
- Off-target edits are permanent
Latest papers
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