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Abstract
A single dose of lonvoguran ziclumeran significantly reduced attack rates in patients with hereditary angioedema.
- Hereditary angioedema is a genetic disorder linked to excessive swelling.
- Lonvoguran ziclumeran is an in vivo CRISPR-Cas9 therapy that targets the KLKB1 gene.
- The treatment demonstrated effectiveness in controlling attack rates during a phase 3 trial.
- Further investigation is needed to address important questions raised by this approach.
Simplified
CRISPR Gene Editing