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Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
Sponsor: Yongguo Yu
Summary
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
Official title: 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
Key Details
Gender
All
Age Range
2 Years - 10 Years
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2025-02-19
Completion Date
2025-09
Last Updated
2025-03-06
Healthy Volunteers
No
Interventions
Dual vector DNA base editor
The base editor is delivered using a dual vector adeno-associated virus (AAV) system and introduced into the child via intrathecal injection to correct the mutated CHD3 gene. The vital signs of the child will be closely monitored during treatment to assess possible acute adverse effects. The child will be followed up regularly after treatment to monitor the success of gene editing and the neurodevelopmental improvement of the child. Possible long-term adverse events will be closely monitored to assess the safety of the treatment.
Locations (1)
Xinhua Hospital affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China