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[HEALTH] · United States · 10 sources

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Part of situation: Prime Medicine in vivo prime editing clinical trials (2 clusters)

Prime Medicine treats first patient with in-body DNA prime editing

prime editing Wilson disease Prime Medicine gene editing ATP7B gene

In a medical milestone published in the journal Nature, scientists have successfully edited human DNA directly inside the body for the first time. Using a high-precision technique known as prime editing, researchers targeted liver cells to correct a genetic mutation responsible for Wilson disease.

Wilson disease is a rare condition caused by mutations in the ATP7B gene, which prevents the body from properly removing excess copper. If left untreated, copper accumulation can lead to severe liver damage, nerve issues, and neurological disorders. Unlike traditional treatments that require lifelong medication, this gene-editing approach aims to provide a one-time, permanent solution.

The treatment, developed by the US-based biotechnology company Prime Medicine, utilizes lipid nanoparticles to deliver the editing machinery directly to hepatocytes. This in vivo method avoids the complex, costly, and invasive process of removing a patient's cells, modifying them in a laboratory, and re-injecting them.

The first patient was dosed on October 5 as part of a Phase 1/2 clinical trial targeting the H1069Q mutation. The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to the PM577 program. Preliminary results from the clinical trial are expected in 2027.

Entities

David R. Liu · FDA · Nature · Prime Medicine · Rhonda Rowland · Wilson disease

Claims

What the coverage asserts, and how many sources carry each claim.

Sources

Bilim dünyasında bir ilk: İnsan DNA'sı vücudun içinde düzenlendi [www.kibrismanset.com]
Τροποποίησαν DNA μέσα στο ανθρώπινο σώμα [odelalis.gr]
Prime Editing Reaches a Living Liver in Wilson Disease [theibulletin.com]
Leber: Gentherapie wird erstmals direkt beim Menschen zur Heilung von Erbkrankheiten eingesetzt [lomazoma.com]
RaillyNews - Historic Milestone: Human DNA Successfully Edited Directly Inside the Body for the First Time [raillynews.com]
İnsan vücudunda ilk kez DNA düzenlemesi yapıldı: Ömür boyu ilaç kullanmaya son verebilir [www.tgrthaber.com]
Bilim dünyasında bir ilk: İnsan DNA'sı doğrudan vücut içinde düzenlendi - Son Dakika Bilim Teknoloji Haberleri [www.cumhuriyet.com.tr]
Για πρώτη φορά τροποποιήθηκε DNA απευθείας εντός του ανθρώπινου σώματος [www.ekriti.gr]
Bilim Dünyasında Çığır Açan Eşik Aşıldı [dokuzeylul.com]
Wereldprimeur: genbewerking herschrijft DNA in levercellen in poging ziekte van Wilson te behandelen | Het laatste binnenlandse en buitenlandse nieuws - upday News [www.upday.com]