One-shot gene therapy aims to tame rett syndrome in young girls
NCT ID NCT06856759
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial tests a single injection of AAV-MECP2 gene therapy into the spinal fluid of 8 girls aged 4-10 with Rett syndrome, a severe neurodevelopmental disorder. The goal is to see if the treatment is safe and can reduce disease severity. The study is not yet recruiting and focuses on finding the right dose.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, 510623, China
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Other studies related to the condition(s) this trial covers.
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