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therapy

The honest summary is that base editing has crossed a genuine and important threshold — from laboratory demonstration to authorised human treatment — without yet becoming a broadly accessible or general-purpose therapy. That second step, extending the approach to more conditions and more patients at a sustainable cost, is likely to be a slower and messier process than the initial scientific breakthrough was.

• First-generation CRISPR: cuts both DNA strands, relies on cellular repair, can produce larger unintended edits.
• Base editing: converts a single DNA letter without cutting both strands, generally produces smaller and more predictable changes.
• Both approaches still require careful off-target screening before and after treatment.
• Base editing is currently better suited to correcting single-letter mutations than to larger insertions or gene rearrangements.