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Gene Editing Platforms
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# Gene Editing Platforms Portal: Genetic Modification Stage: Clinical and emerging Evidence: Clinical practice Template: Technology Risk: High Reversibility: Difficult to reverse Last reviewed: Jun 2026 == Summary == CRISPR, base editing, prime editing, and epigenetic editing expand what medicine can rewrite, regulate, or silence. == Key takeaways == * The editing enzyme is only half the product; delivery determines where it can work. * Ex vivo editing is easier to control than in vivo editing, but less convenient. * Future aging applications will depend on targets with clear risk-benefit math. == Platform map == Nuclease editing cuts DNA. Base editing changes individual letters without a double-strand break. Prime editing can write more flexible edits. Epigenetic editing can tune gene expression without altering the underlying sequence. These platforms are not interchangeable. Each has a different error profile, payload size, delivery constraint, and reversibility model. == Human deployment == Ex vivo workflows edit cells outside the body and return them after quality control. In vivo workflows deliver editors directly into tissues, which can be more scalable but harder to recall. For future human enhancement, the barrier is not merely technical capability. Durable edits for low-risk conditions require extremely high confidence because harms can be permanent. == Open questions == * Which editing method fits which disease and delivery route? * How is durability of an edit confirmed over a lifetime? == Watchlist == * Lipid nanoparticles * AAV capacity * Off-target assays * Epigenetic reversibility == References == * FDA CRISPR therapy approval — U.S. FDA CASGEVY approval materials. https://www.fda.gov/vaccines-blood-biologics/casgevy. Regulatory source for approved ex vivo CRISPR therapy indications. * Exa-cel clinical evidence — Frangoul et al., New England Journal of Medicine, 2024. https://pubmed.ncbi.nlm.nih.gov/38661449/. Clinical outcomes source for exagamglogene autotemcel in severe sickle cell disease. == Categories == [[Category:Genetic Modification]] [[Category:CRISPR]] [[Category:base editing]] [[Category:delivery]]