Clinical Trial

Gene Editing as a Therapeutic Approach for Rett Syndrome

Study acronym: MECPer-3D
Recruiting
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Record status
This record was last updated August 20, 2025 (before its estimated November 1, 2025 completion). Its status may not reflect the trial's current state.
Summary
We designed the project to validate CRISPR/Cas9-based gene editing combined with AAV-based delivery for correction of the most common MECP2 mutations both in vitro and in vivo.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2023-02-13.
Trial Details
NCT Number NCT05740761
Lead Sponsor University of Siena
Conditions Rett Syndrome
Enrollment 40 participants
Start Date 2021-03-01
Primary Completion 2025-11-01 (estimated)
Study Completion 2026-03-01 (estimated)
Updated on ClinicalTrials.gov 2025-08-20