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In Vivo Gene Editing and Delivery
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# In Vivo Gene Editing and Delivery Portal: Genetic Modification Stage: Clinical, first approvals landed Evidence: Clinical practice Template: Technology Risk: Moderate Reversibility: Irreversible Last reviewed: Jun 2026 == Summary == Editing genes inside the body has moved from concept to approved therapy, and delivery — not the editor — is now the dominant constraint on what can be treated. == Key takeaways == * In vivo editing delivers the editor into the patient rather than editing cells in a lab and reinfusing them. * Delivery vehicles — lipid nanoparticles and viral vectors — determine which tissues are reachable. * The first approvals prove the concept; broad tissue reach remains the frontier. == Editor versus delivery == The editing tools — CRISPR nucleases, base editors, prime editors — are increasingly precise. The harder problem is getting them into the right cells at the right dose without triggering immunity or hitting the wrong tissue. Lipid nanoparticles excel at reaching the liver, which is why early in vivo successes cluster around liver-expressed targets. Viral vectors such as AAV reach other tissues but carry payload-size limits and immune considerations. == From proof to practice == An in vivo CRISPR therapy for transthyretin amyloidosis showed durable knockdown of a disease protein after a single infusion, demonstrating that systemic editing could work in people. Ex vivo edited cell therapies for sickle cell disease and beta-thalassemia then reached regulatory approval. The next expansion depends on delivery: reaching muscle, brain, and other tissues safely, controlling dose, and managing the fact that most editing is permanent. Redosing, off-target surveillance, and long-term follow-up are the practical agenda. == Open questions == * Which delivery advances unlock tissues beyond the liver? * How is long-term safety monitored when edits are permanent? == Watchlist == * Non-liver delivery * Base and prime editing in vivo * Off-target long-term surveillance == References == * In vivo CRISPR editing for transthyretin amyloidosis — Gillmore et al., NEJM, 2021. https://www.nejm.org/doi/full/10.1056/NEJMoa2107454. First clear demonstration of systemic in vivo CRISPR editing in humans. * FDA approves first CRISPR gene therapy (Casgevy) — U.S. FDA, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease. Approval of exagamglogene autotemcel for sickle cell disease. == Categories == [[Category:Genetic Modification]] [[Category:CRISPR]] [[Category:delivery]] [[Category:LNP]] [[Category:AAV]]