简单说明
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.
深入了解
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
- New England Journal of Medicine · 2021
CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis ↗