Gene Editing as a Therapeutic Approach for Rett Syndrome

Sponsor
University of Siena
Study ID
NCT05740761
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
FEMALE
Age
6 Months - N/A
Healthy Volunteers
Not accepted

Interventions

  • Gene editing in vitro — OTHER
    Testing of gene editing efficiency in vitro in human cellular models derived from patients

Study Details

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.

Key Dates

Start date
Mar 1, 2021
Status verified
Aug 2025
Primary completion
Nov 1, 2025
Completion
Mar 1, 2026

Study Design

Enrollment
40 participants (estimated)

Primary Outcome Measure

Editing efficiency [ Time Frame: 3 years ]

Central Contacts

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