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Gene Editing as a Therapeutic Approach for Rett Syndrome

NCT05740761 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 40

Last updated 2025-08-20

No results posted yet for this study

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.

Conditions

Interventions

OTHER

Gene editing in vitro

Testing of gene editing efficiency in vitro in human cellular models derived from patients

Sponsors & Collaborators

  • University of Siena

    lead OTHER

Eligibility

Min Age
6 Months
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-01
Primary Completion
2025-11-01
Completion
2026-03-01

Countries

  • Italy

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05740761 on ClinicalTrials.gov