gene editing

Fatal Outcome in Single-Patient Gene Editing Trial Raises Safety Concerns

A 6-year-old girl died seven days after receiving a personalised base-editing therapy for a rare genetic disorder in an unregistered Chinese trial, prompting international concern about oversight.

First-in-Human CRISPR-Cas9 Editing of ANGPTL3 Shows Dose‑Dependent Protein Knockdown with Acceptable Short‑Term Safety in Phase 1 Trial