Gene Editing & Genetic Medicine
Therapies that directly and durably modify or silence disease-associated genes inside the body (in vivo base editing, CRISPR-style editing) to produce a one-time treatment effect, as opposed to a drug requiring lifelong repeated dosing.
Also known as: gene therapy, gene editing, genterapi, genbehandling, genredigering, base editing, in vivo gene editing, CRISPR, PCSK9
Part of: Health, Aging & Longevity
How authors use it
Oldest first (extraction date when no publication date is known).
| Author | Their term | Their meaning |
|---|---|---|
| Én enkelt behandling kan gi svært langvarig kolesterolsenkning: – Noe radikalt nytt Bård Amundsen · 2026-05-29 | PCSK9 | A liver gene whose protein product raises blood LDL-cholesterol; the article treats 'turning off PCSK9' as more or less equivalent to 'lowering LDL', without elaborating the receptor-recycling mechanism. |
Documents (1)
- Én enkelt behandling kan gi svært langvarig kolesterolsenkning: – Noe radikalt nytt Bård Amundsen · 2026-05-29 source ↗ Reader 🖥️
A single infusion of an in-vivo gene-editing therapy that permanently disables the PCSK9 gene produced deep and durable (18-month) LDL-cholesterol reduction in a small first-in-human trial, suggesting that cardiovascular disease prevention may shift from lifelong daily medication toward a one-time, vaccine-like treatment.
Propositions touching this concept (1)
By publication month, oldest first (extraction month when no publication date is known).
2026-05
- explicitThe treatment's developers intend for it to become an affordable, primary-care preventive treatment for millions, not another costly niche gene therapy.
Open gaps (2)
- incompleteA 35-patient trial with an 18-month observation window is treated as sufficient basis for headline framing ('radically new', 'a vaccine'), without discussing statistical power, control-group design (is this placebo-controlled, dose-ranging, open-label?), or how many of the 35 patients were in the highest-dose (62%-reduction) group versus lower, less dramatic doses.Én enkelt behandling kan gi svært langvarig kolesterolsenkning: – Noe radikalt nytt — read next: The original NEJM paper (Vafai et al., 'In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia') for trial design details, and later Phase 2/3 results from the planned ~200-patient study.
- incompleteThe article treats 'off PCSK9' and 'lower LDL' as essentially interchangeable outcomes and never addresses whether a permanent, irreversible gene edit carries different risk than the existing reversible PCSK9-inhibitor drugs it is being compared to — e.g., what happens if a patient develops an adverse reaction years later with no way to 'undo' the edit, unlike stopping a pill or antibody injection.Én enkelt behandling kan gi svært langvarig kolesterolsenkning: – Noe radikalt nytt — read next: Look for expert commentary (bioethics, gene-therapy safety literature) specifically on irreversibility as a distinct risk category from reversible pharmacological intervention, rather than just 'will there be side effects'.