ACTIVE: AAV-MECP2 gene-therapy study – China

Status: Active, not recruitingStudy: Single-dose AAV-MECP2 safety, tolerability and efficacy study
Registry number: NCT06856759
Location: Guangzhou Women and Children’s Medical Center, Guangzhou, China
Participants: Females aged 4–10 years

What is being studied?
This small early-stage study is assessing the safety, tolerability and preliminary effects of a single-dose AAV-based MECP2 gene therapy in eight girls with Rett syndrome. Participants are followed for safety and clinical outcomes.

The registry lists the study as active but not currently recruiting.

Official study record: https://clinicaltrials.gov/study/NCT06856759

Information checked 3 September 2026. Gene therapy remains experimental and can involve serious or unknown risks. The official registry and treating Rett specialist should be consulted for updates.