Actively Recruiting
Gene Editing as a Therapeutic Approach for Rett Syndrome
Led by University of Siena · Updated on 2025-08-20
40
Participants Needed
1
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Gene Editing as a Therapeutic Approach for Rett Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Female patients diagnosed with Rett Syndrome confirmed genetically by NGS with one of these MECP2 mutations: c.473C>T (p.T158M), c.502C>T (p.R168X), c.763C>T (p.R255X), or c.916C>T (p.R306C)
- Age above 6 months
- Parents or legal guardians able to provide informed consent for participation
You will not qualify if you...
- NGS diagnosis showing normal MECP2 gene sequence
- Presence of MECP2 mutation different from those studied
- Parents or legal guardians unwilling to provide informed consent for participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University of Siena
Siena, Siena, Italy, 53100
Actively Recruiting
Research Team
I
Ilaria Meloni, BS.PhD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here