Gene Editing as a Therapeutic Approach for Rett Syndrome
Recruiting
Observational Study
Rett Syndrome
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 6 Months and older
- Sex
- Female
- Study type
- Observational
- Participants needed
- 40 (estimated)
- Sponsor
- University of Siena · Other
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Patients -exclusively female- since the pathology is linked to the X chromosome, with a clinical diagnosis of Rett syndrome confirmed at the genetic level by the identification, through NGS analysis, for one of the recurrent mutations (mutational hotspots) in the MECP2 gene ob…
Contacts