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New England Journal of Medicine2021Gillmore et al.The first report of CRISPR administered into a human bloodstream to edit a gene inside the body.
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.
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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.
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