Gene Editing as a Therapeutic Approach for Rett Syndrome
ConditionRett Syndrome
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexFemale
Age6+
SponsorUniversity of Siena
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.
Eligibility criteria
Qualifiers
Age above 6 months;
Availability of parents or legal guardians to provide free and informed consent to participate in the study
Disqualifiers
NGS diagnosis with the normal outcome;
Positive NGS diagnosis for mutation in MECP2 but with the presence of a mutation different from those under study.
Unwillingness of parents or legal guardians to provide free and informed consent to participate in the study;
Trial design
Treatments tested in this trial
- Gene editing in vitro
Treatment groups
No treatment groups listed
Sponsors and collaborators
Source ClinicalTrials.gov