Status: Recruiting
Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
To evaluate the safety, tolerability and preliminary efficacy study of a single intrathecal injection of the dual vector AAV-CHD3-R1025W base editor for the treatment of developmental disorders caused by the R1025W mutation in the CHD3 gene
Participants needed: 1
Trial details
Phase: Early Phase 1Age: 2-10Biological sex: AllType: InterventionalSponsor: Yongguo YuUpdated: Mar 6, 2025Locations: 1
Eligibility criteria
Clinical diagnosis of Snijders Blok-Campeau syndrome [+3]
Brain tumor or intracranial space-occupying lesion [+8]