One-shot gene editor aims to correct a brain disorder at its source
⭐️ CURE ⭐️
Terminated
This trial tests whether a single injection of a gene-editing tool can safely correct a specific mutation in the CHD3 gene that causes Snijders Blok-Campeau syndrome, a condition marked by developmental delay and intellectual disability. The gene editor is delivered directly into…
Phase: EARLY_PHASE1 • Sponsor: Yongguo Yu • Aim: ⭐️ CURE ⭐️
Last updated Aug 01, 2026 00:00 UTC