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categories: ["genetics", "bodies"]categories: ["genetics", "bodies"]tags: ["gene therapy", "rare disease", "aav", "drug pricing", "clinical trials", "cell therapy"]tags: ["gene therapy", "rare disease", "aav", "drug pricing", "clinical trials", "cell therapy"]summary: "Genetic modification of a living person's body cells to treat disease, producing a change that is not passed on to their children."summary: "Genetic modification of a living person's body cells to treat disease, producing a change that is not passed on to their children."updated: "2026-10-29"updated: "2026-11-01"humanEvidence: "Licensed products treat inherited retinal dystrophy, sickle cell disease and spinal muscular atrophy in people; durability varies, and clotting-factor expression after haemophilia gene therapy declines over the years after treatment."humanEvidence: "Licensed products treat inherited retinal dystrophy, sickle cell disease and spinal muscular atrophy in people; durability varies, and clotting-factor expression after haemophilia gene therapy declines over the years after treatment."access: "Approved in the US and Europe for a short list of rare diseases and blood cancers, delivered at a small number of specialist centres; list prices run from roughly $400,000 to $4.25 million per patient."access: "Approved in the US and Europe for a short list of rare diseases and blood cancers, delivered at a small number of specialist centres; list prices run from roughly $400,000 to $4.25 million per patient."reversibility: "context"reversibility: "context"lines 101–106 → 101–10786 unchanged lines not shown
- [[governance-of-genome-editing]]- [[governance-of-genome-editing]]- [[access-and-inequality]]- [[access-and-inequality]]- [[crispr-off-target-effects]]- [[crispr-off-target-effects]]- [[in-vivo-car-t]] ## References## References removed, struck through added, underlinedLine numbers count the serialised markdown of each revision, frontmatter included.
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