PubMed · 2026-05-13
Scientists used a precise gene-editing tool called a base editor to correct a genetic mutation in immune cells, successfully treating a rare and dangerous inflammatory disease in mice without needing to cut both strands of DNA. The study also found that different editing platforms and cell types carry distinct safety risk profiles.
Cytosine base editing achieved 62–89% efficiency in correcting the target mutation across fibroblasts, T cells, and blood stem cells.
Transplantation of edited blood stem cells protected mice from virus-triggered hyperinflammation, validating the therapeutic approach.
Hyperactive base editors caused broader off-target DNA changes and more structural variants than standard CRISPR-Cas9, with stability of chromosomal rearrangements varying by cell type.