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Research paper · Clinical

CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis

The first report of CRISPR administered into a human bloodstream to edit a gene inside the body.

Simple explanation

Until this paper, gene editing in patients meant taking cells out, editing them, and putting them back. This showed CRISPR delivered by infusion, editing a gene inside people's livers, and reducing the harmful protein by a large margin.

Go deeper

Gillmore and colleagues reported the first-in-human study of NTLA-2001, a lipid-nanoparticle CRISPR-Cas9 therapy targeting hepatic TTR, showing dose-dependent serum transthyretin reduction after a single infusion with mild adverse events at the doses studied.

Sources

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

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Diseases

ATTR Amyloidosis

Treatments

Nexiguran ziclumeran (nex-z)
Educational information only This page is a reference, not medical advice. Research and regulatory status change; check the last-updated date above and confirm anything important against the primary sources listed.