RECRUITING: MECP2 gene-editing laboratory research – Italy

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Status: RecruitingProject: Gene editing as a therapeutic approach for Rett syndrome
Registry number: NCT05740761
Location: University of Siena, Italy
Participants: Females aged over 6 months with one of four specified recurrent MECP2 variants

What is being studied?
Researchers are collecting participant samples for laboratory work testing CRISPR/Cas9 gene editing combined with AAV delivery. The aim is to investigate correction of selected common MECP2 variants in laboratory and preclinical models.

This is observational sample-based research, not administration of gene editing to participants.

Contact: Dr Ilaria Meloni — +39 0577 233259 — ilaria.meloni@dbm.unisi.it
Official study record: https://clinicaltrials.gov/study/NCT05740761

Information checked 3 September 2026. Participation criteria are mutation-specific. Contact the research team for the current protocol and consent information.