Condition: Rett Syndrome · Sponsor: University of Siena
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.
This description comes directly from the study's public registry record.
Ilaria Meloni, BS.PhD · +390577233259 · ilaria.meloni@dbm.unisi.it
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| University of Siena | Siena, Siena, Italy | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05740761