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Gene Editing as a Therapeutic Approach for Rett Syndrome — MECPer-3D  

Rett Syndrome Clinical Trial

Official title:
Personalized MECP2 Gene Therapy Using CRISPR/Cas9 Technology Coupled to AAV-mediated Delivery in 3D Cell Culture and KI Mice

Clinical Trial 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.

Clinical Trial Description

The project aims 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. The laboratory of the principal investigator is an active member of the European Reference Network for rare malformation syndromes and rare intellectual and neurodevelopmental disorders (ERN-ITHACA). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05740761
Study type Observational
Source University of Siena
Contact Ilaria Meloni, BS.PhD
Phone +390577233259
Email ilaria.meloni@dbm.unisi.it
Status Recruiting
Phase
Start date March 1, 2021
Completion date March 1, 2024
View Details
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