Condition: Developmental Delay Disorder · Intellectual Disability · Rare Diseases · Sponsor: Yongguo Yu
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
This description comes directly from the study's public registry record.
Xiaomei Luo, Ms., Master · +86-25-25076466 · luoxiaomei@shsmu.edu.cn
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| Xinhua Hospital affiliated to Shanghai Jiao Tong University School of Medicine | Shanghai, Shanghai Municipality, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06860672