Ignite Creation Date:
2025-12-24 @ 12:18 PM
Ignite Modification Date:
2025-12-27 @ 9:27 PM
Study NCT ID:
NCT05740761
Status:
RECRUITING
Last Update Posted:
2025-08-20
First Post:
2023-02-13
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Editing as a Therapeutic Approach for Rett Syndrome
Sponsor:
University of Siena
Organization:
Study Overview
Official Title:
Personalized MECP2 Gene Therapy Using CRISPR/Cas9 Technology Coupled to AAV-mediated Delivery in 3D Cell Culture and KI Mice
Status:
RECRUITING
Status Verified Date:
2025-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
MECPer-3D
Brief 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.
Detailed 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 Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: